Oncology orphan drug production. Medical search. Frequent questions

The FDA has granted PTC124 Subpart E designation for expedited development, evaluation and marketing and has granted Orphan Drug designations for the treatment of CF and DMD due to nonsense mutations. (cff.org)
Nirogacestat, the drug in testing against desmoid tumors, is undergoing Phase 3 study, and has received Orphan Drug Designation and Fast Track Designation from the FDA. (cryptoculture.info)
AstraZeneca and Merck & Co., Inc., Kenilworth, N.J., US (Merck: known as MSD outside the US and Canada) announced that they were granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for Lynparza (olaparib) for the treatment of pancreatic cancer. (worldpharmanews.com)
The FDA granting Orphan Drug Designation is a positive step for patients with pancreatic cancer and continues to reinforce the importance of our collaboration in bringing Lynparza to more patients in need. (worldpharmanews.com)
The FDA selectively grants Breakthrough Therapy Designation to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition, and preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). (bigboardalerts.com)
We are excited about the potential therapeutic benefit of felzartamab through this Breakthrough Therapy Designation by the FDA, following the Orphan Drug Designation received in May," said Dr. Andrew Zhu , President of I-Mab. (bigboardalerts.com)
The post Felzartamab Granted Breakthrough Therapy Designation by U.S. Food and Drug Administration (FDA) for Primary Membranous Nephropathy (PMN) appeared first on Pharma Journalist . (bigboardalerts.com)
The U.S. Food and Drug Administration has awarded Rare Pediatric Disease Designation (RPDD) for diffuse intrinsic pontine glioma (DIPG) and Orphan Drug Designation for treatment of malignant glioma to OKN-007, an investigational drug discovered at the Oklahoma Medical Research Foundation and being developed by Oblato, Inc. (news-medical.net)
The FDA granted Breakthrough Therapy designation and Priority Review for REZUROCK and reviewed the New Drug Application (NDA) under the Real-Time Oncology Review (RTOR) pilot program. (yahoo.com)
Emmaus' SCD therapy has Orphan Drug designation in the U.S. and Europe and Fast Track designation from the FDA. (emmausmedical.com)
Ionis-HTTRx has been granted orphan drug designation by the US Food and Drug Administration and by the European Medicines Agency. (medscape.com)

Orphanet, the portal for rare diseases and orphan drugs. (wikipedia.org)
There are of course other treatments besides orphan drugs for these diseases. (atheneum.ai)
Understanding these regulatory nuances can be critical for the development of such medicinal products for rare diseases, also known as orphan medicinal products. (xtalks.com)
Furthermore, the presentation aims to provide the audience with an understanding of how certain legal tools and incentives have stimulated the growth of the drug products for rare diseases and what other things can be done in order to achieve a better impact. (xtalks.com)
Despite these efforts HTA SA is not routinely integrated into the clinical development of new medicines and it faces new challenges with the development of advanced therapy medicinal products, stratified medicines and medicines used in orphan diseases. (ohe.org)
The firm also notes that proposed new legislation on healthcare provision currently making its way through Russia's Duma (parliament) includes establishment of a legal recognition for orphan diseases and the drugs used to treat them, with provisions for reimbursement of treatments for orphan diseases, at both regional and federal level. (pharmatimes.com)
The bill, which reportedly defines orphan diseases as those with no more than 10 patients out of a 100,000 population, also includes proposals for a system for the licensing and procurement of orphan drug products. (pharmatimes.com)
AOP Orphan thus continues its consistent growth trajectory towards being a pan-European health care group with a focus on rare and complex diseases. (aop-health.com)
The AOP Orphan Group is the European pioneer in the field of special diseases with a complex management. (aop-health.com)
We try to look beyond this: Research and development process leading to such orphan drugs also provides valuable insights into the natural history of the disease investigated and may even offer insights into causes and possible therapeutic options of far more common diseases. (biovalley.ch)
He has strong translational medicine and strategic experience across rare diseases, fibrosis, immunology, and oncology. (biovalley.ch)
Thomas is an internationally recognized scientist with track record in clinical research of neuromuscular and neuro-ophthalmology orphan diseases and has hands-on experience in the regulatory process of orphan disease product registration. (biovalley.ch)
Company develops a new class of protein interaction inhibitors to tackle drug resistance in cancer and to cure other rare diseases. (biovalley.ch)
I-Mab is evaluating felzartamab in oncology and autoimmune diseases. (bigboardalerts.com)
And yet, interest (and therefore investment) in this field of pharmaceuticals has fluctuated over time, with oncology and orphan diseases now seeing significant cashflow. (leadersleague.com)
A global biopharmaceutical company on a number of transactions, including a global strategic collaboration to discover, develop and commercialize a pipeline of innovative small molecule therapeutics against certain oncology and immunology targets and an exclusive worldwide option and license agreement for a costimulation antagonist for autoimmune and inflammatory diseases. (ropesgray.com)
If we consider orphan drugs (products for rare diseases), price levels can be over $500,000 per year, higher than the cost of the median home, the largest purchase for most people. (pharmexec.com)

SUZHOU, China , June 6, 2023 /PRNewswire/ - Alphamab Oncology (stock code: 9966.HK) announced that updated data from the phase II clinical study of the chemo-free regimen of KN026 in combination with KN046 (KN026-203) were presented at the 2023 American Society of Clinical Oncology (2023 ASCO) Annual Meeting. (weeklyreviewer.com)

The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is done. (terrapinn.com)

With the biopharma industry's continuous moved toward drugs targeting smaller patient groups, such as highly targeted oncology therapeutics, personalized medicines and orphan drugs, NIBRT spotted the growing demand for advanced technology and robust aseptic manufacturing that is more efficient and cost-effective. (pharmaceuticalcommerce.com)
SpringWorks Therapeutics (SWTX) The first Goldman pick we're looking at is a clinical-stage biotech firm in the oncology niche. (cryptoculture.info)
Stefano is Chief Executive Officer at AZAFAROS B.V. He is an accomplished biopharma executive with over 20 years of experience and a string of successes in launch and growth of orphan medicines and specialty therapeutics. (biovalley.ch)
Precision Therapeutics (NASDAQ:AIPT) operates in two business areas: first, applying artificial intelligence to personalized medicine and drug discovery to provide personalized medicine solutions for clients in the pharmaceutical, diagnostic, and biotech industries, and second, production of the FDA-approved STREAMWAY® System for automated, direct-to-drain medical fluid disposal. (globenewswire.com)
Lipigon Pharmaceuticals has entered a development and license agreement for its clinical-stage drug candidate Lipisense with Leaderna Therapeutics. (nordiclifescience.org)

Notably, the FDA has granted approval to a greater number of gene therapy drugs in the latter half of 2022 alone compared to the prior five-year period. (wowktv.com)
Initial results from the pre-planned interim analysis were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting. (advfn.com)
Based on the positive phase 3 data, Stemline, with the support of Radius, submitted a New Drug Application (NDA) in June 2022 to the FDA. (menarini.gr)

American Society of Clinical Oncology educational book. (wustl.edu)

In September 2021, GeoVax expanded its immuno-oncology pipeline and added a new technology platform through the acquisition of exclusive rights to Gedeptin ® , a novel patented product for the treatment of solid tumors through a gene therapy strategy known as GDEPT (Gene-Directed Enzyme Prodrug Therapy). (gabio.org)

Pricing orphan products, especially one-time treatments, is complex and a key element to ensuring timely access for rare disease patients. (terrapinn.com)
Russia's proposed list of "vital and essential" drugs for 2012 includes a number of innovative cancer treatments. (pharmatimes.com)
Analysts at IHS Global Insight comment that the expansion of the list to include so many innovative drugs, particularly in oncology, is testament to the Russian authorities' increasing willingness to invest more in reimbursement of medicines, including those which are innovative and expensive, although they add that coverage for these treatments continues to be somewhat limited across the Russian Federation. (pharmatimes.com)
Stemline is focused on bringing transformational oncology treatments to cancer patients, and currently commercializes a novel targeted treatment for patients with blastic plasmacytoid dendritic cell neoplasm in both the United States and Europe. (menarini.gr)
The TumorGenesis PDx model will initially be developed for three orphan cancers, Multiple Myeloma, Triple-Negative Breast cancer (TNBC) and Ovarian cancers, all of which are areas that have a high unmet need for new and effective treatments that are tailored to patients' unique tumor profiles. (globenewswire.com)

If these results are reproduced in the second Phase 3 study and are supported by the results of a planned interim analysis of the Phase 3 extension study, the company expects to submit a New Drug Application with the U.S. Food and Drug Administration in the first quarter of 2017. (prnewswire.com)

These lab-made proteins, designed to simultaneously target two or more antigens, promise to revolutionize treatment methods for a range of medical conditions, particularly in oncology and. (reportlinker.com)

Today s pipeline drugs are the medicines that companies will be launching and marketing for the next 10 to 20 years, he says. (pharmamanufacturing.com)
AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients' lives and the Company's future. (worldpharmanews.com)
With at least six new medicines to be launched between 2014 and 2020 and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as a key growth driver focused on lung, ovarian, breast and blood cancers. (worldpharmanews.com)
US outfit The Medicines Company has been valued by Swiss pharmaceuticals giant Novartis at $9.7 billion - largely, it seems, on the basis of one drug. (leadersleague.com)

AstraZeneca and Merck & Co have entered a global strategic oncology collaboration to co-develop and co-commercialise AstraZeneca's Lynparza (olaparib) for multiple cancer types. (nordiclifescience.org)

SCD is an orphan disease in the U.S affecting approximately 100,000 patients in the U.S and millions worldwide with significant unmet medical needs. (emmausmedical.com)

As with Nirogacestat, the FDA has given Orphan Drug and Fast Track designations to this program. (cryptoculture.info)

AOP Orphan Pharmaceuticals AG (AOP Orphan) took over the Viennese pharmaceutical company Amomed and the Luxembourgish health-tech company SciPharm in September 2020. (aop-health.com)
In the third quarter of 2020, AOP Orphan took over the health care companies Amomed Pharma GmbH and SciPharm S.à.r.l. (aop-health.com)

Pharma is facing wide-ranging issues from consolidation, weak pipelines, longer regulatory review, fewer years of commercial intellectual property protection, generics, price pressures and political heat, he says.Oncology is one therapeutic area that s moving closer to the vision of personalized medicine. (pharmamanufacturing.com)

NEW YORK, NY / ACCESSWIRE / July 16, 2021 / Kadmon Holdings, Inc. (Nasdaq:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has approved REZUROCK ™ (belumosudil) 200 mg once daily (QD) for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. (yahoo.com)
The FDA approved this NDA six weeks ahead of the Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2021. (yahoo.com)

The evolving landscape of oncology is introducing significant advancements, with CAR-T therapy marking a prominent position in revolutionizing the approaches taken to malignancy management. (reportlinker.com)
The preclinical and clinical data on PTC124 support our hope that this drug will be an important disease-modifying therapy for cystic fibrosis," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. (cff.org)
Due to the strategic focus on providing holistic therapy solutions which include drugs, medical devices and patient services and an innovative product pipeline, we are convinced that this positive development will come to fruition," Steiner continues. (aop-health.com)
We] see upside to the commercial outlook for SWTX's rare oncology programs driven by extended duration of therapy, but view the clinical results expected this year as well-understood and therefore unlikely to significantly drive stock performance. (cryptoculture.info)
In 2018, Novartis bought Advanced Accelerator Applications, gene therapy biotech companies AveXis and CELLforCURE, and Endocyte, a pharma firm specialising in drug conjugations (drugs that work by combining with other molecules in the body). (leadersleague.com)
This follows a license agreement with SyntArray, LLC, announced on March 13th , and is the latest milestone in the Company's strategy to bring together ground-breaking technologies to develop the next generation of patient derived ("PDx") tumor models for precision cancer therapy and drug development. (globenewswire.com)
This model is expected to create more accurate results when testing drugs for personalized therapy and when developing new drugs, compared to testing with traditional animal or cell culture models. (globenewswire.com)

The conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies. (terrapinn.com)
What are the current roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production? (terrapinn.com)
The Contract Development and Manufacturing Organization (CDMO) sector for advanced therapeutic drugs, such as gene and cell therapies, is currently undergoing significant transformation. (reportlinker.com)
As a result, we hear that an age of personalized medicine is dawning, in which therapies will be tailored to groups of patients, or even individuals.This brave new world would mean the end of the blockbuster drug model and would transform the way that medicine is practiced, and drugs are made and marketed. (pharmamanufacturing.com)
The manufacturing response In response to the promises and threats of personalized therapies, pharmaceutical companies must accelerate retooling and streamlining of production lines, just as heavy and light manufacturers in other industries have done over the last twenty years. (pharmamanufacturing.com)
By developing innovative therapies and a broader territorial presence, we are convinced that we will continue to generate significant growth," says Andreas Steiner , CEO of AOP Orphan. (aop-health.com)
The takeover of Amomed and SciPharm brings the research and development of drugs as well as the development of medical devices and patient services together under one roof, creating optimal conditions for developing integrated therapies. (aop-health.com)
Therapies for treating glioblastoma brain cancer can be delivered with greater precision and existing drugs can be used in new ways. (news-medical.net)

The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. (terrapinn.com)
As a dynamic life-science entrepreneur he currently serves on the Board of three biotech companies with orphan disease product pipelines and is managing partner of Viopas Venture Consulting. (biovalley.ch)

When looking at the top selling drugs in 2006 vs. 2016, we can see a large increase in the number of biologics as well as specialty products. (pharmexec.com)

After the takeover, the health care group will concentrate on four therapeutic divisions: hemato-oncology, cardiology & pulmonology, neurology & metabolic disorders, and intensive care medicine. (aop-health.com)

US-based insurers and single payer agencies around the world have a lot of impact of access and reimbursement for orphan products. (terrapinn.com)

Dr. Wen is the Director of the Center for Neuro - Oncology at Dana Farber Cancer Institute, a Professor of Neurology at Harvard Medical School and is a member of the Board of Directors and immediate past President of the Society for Neuro - Oncology. (cnspharma.com)

He has knowledge on a range of orphan oncology indications including Merkel Cell Carcinoma and is able to discuss the challenges around orphan drug patient journeys. (atheneum.ai)

What are the best commercial strategies for orphan products? (terrapinn.com)
What are the current best practices for pricing orphan products, negotiating innovative contracts and working with payers around the world? (terrapinn.com)
Biopharma companies at all stages of development, rely on our expertise and broad capabilities to produce the clinical trial supply of drug products they need, when they need them. (cambrextallinn.info)
SP Scientific Products (SP), a provider of fill-finish drug manufacturing services, is collaborating with the National Institute for Bioprocessing Research and Training (NIBRT) in Dublin, Ireland, to install a modular multi-container filling line and isolation system to further augment training offerings in aseptic biopharma fill-finish operations. (pharmaceuticalcommerce.com)
The initial stage of the study is being funded by the FDA pursuant to its Orphan Products Clinical Trials Grants Program. (gabio.org)
With 18 production sites and 9 Research and Development centers, Menarini's products are available in 140 countries worldwide. (aap.com.au)
It is also "no coincidence," they add, that the increasing numbers of companies whose products are appearing on the essential drugs list are choosing to establish production and/or development operations in Russia. (pharmatimes.com)
Regarding the Gedeptin program, Mr. Dodd stated, "We expect to soon complete the first stage of this study, which is being funded in part by the U.S. Food and Drug Administration (FDA) pursuant to its Orphan Products Clinical Trials Grants Program, and then initiate an expanded Phase 2 trial focused on expedited FDA registration. (geovax.com)
Mr. Dodd said, "Our progress in developing a continuous avian cell line system is nothing short of transformative for manufacturing our MVA-based vaccines and immunotherapies, and we recently partnered with Advanced Bioscience Laboratories, Inc. to support the production of our MVA-based products through late-stage development and eventual commercialization. (geovax.com)
Stay current on our latest innovations, products, and science news in our weekly Drug Discovery and Development Email Newsletter. (discoverx.com)

Understanding the regulatory and clinical landscape for rare disease drug development. (terrapinn.com)
Drug substance tech transfer sounds so simple on the surface, but it's a complex activity carrying a number of technical, regulatory, and quality risks. (cambrextallinn.info)
Our teams of creative problem-solvers work closely with you to accelerate drug development timelines, optimize processes, and meet regulatory requirements. (cambrextallinn.info)
In addition to industry appointments, he was previously an adjunct professor in the Drug Regulatory Program at LIU. (xtalks.com)
The regulatory review for elacestrant is also underway in the US as the Food and Drug Administration (FDA) has recently accepted a new drug application for elacestrant designating a priority review. (aap.com.au)
Investors are attracted to the rare disease/orphan drug sector in part because it offers certain regulatory incentives and typically has exceptional revenue growth rates. (biovalley.ch)

We have a collaboration and asset purchase agreement with Reata Pharmaceuticals for all trial data and drug manufacturing documentation. (cnspharma.com)

As the rare cancer field evolves and diagnosis becomes more precise, how can all stakeholders work together to advance drug development for rare cancer patients? (terrapinn.com)
Others see the blockbuster model as outdated, since even the most successful drugs don t work in all the patients all the time. (pharmamanufacturing.com)
However, once patients were singled out and treated who expressed the HER2/neu gene, linked to growth of more aggressive tumors, the drug s efficacy shot up, justifying the adverse events. (pharmamanufacturing.com)
This is an exciting potential new treatment for patients afflicted with nonsense-mutation-mediated CF. We look forward to the next stage of clinical development to demonstrate the benefits of this promising new investigational drug. (cff.org)
Consistent with the prior clinical study of fostamatinib in ITP, this FIT Phase 3 study demonstrated that fostamatinib provided a robust and enduring benefit for those patients who responded to the drug candidate. (prnewswire.com)
Patients are connected to the doctors treating them by an app which enables the dose of the drug which is administered using a pump to be controlled - precisely and tailored to the individual patient. (aop-health.com)
Updated clinical data from the Phase 1/2 study sponsored by GSK plc (LSE/NYSE: GSK) evaluating nirogacestat in combination with low-dose belamaf (belantamab mafodotin-blmf), GSK's antibody-drug conjugate targeting BCMA, in patients with RRMM will be presented in a poster presentation. (advfn.com)
We believe that nirogacestat has the potential to be a significant advance for patients and we look forward to our continued discussions with the FDA as they review our New Drug Application. (tmcnet.com)
A practice-changing study, NRG Oncology clinical trial NRG-RTOG 9802, has demonstrated, for the first time, a survival benefit of adjuvant chemotherapy following radiotherapy over radiotherapy alone in certain subgroups of patients with high-risk, low-grade glioma (WHO classification: LGG, grade II), a type of brain tumor that originates from glial cells. (news-medical.net)
In the first human trial, an experimental antisense drug (Ionis-HTTRx, Ionis Pharmaceuticals) successfully lowered the level of mutant huntingtin protein (mHTT) in spinal fluid of patients with Huntington's disease , researchers reported today. (medscape.com)

The company has an active pipeline, with programs investigating drug candidates for the treatment of desmoid tumors, plexiform neurofibromas, multiple myeloma, and metastatic solid tumors. (cryptoculture.info)

A Ludwig Cancer Research study has dissected how radiotherapy alters the behavior of immune cells known as macrophages found in glioblastoma (GBM) tumors and shown how these cells might be reprogrammed with an existing drug to suppress the invariable recurrence of the aggressive brain cancer. (news-medical.net)

PTC124 is an orally delivered investigational new drug in Phase 2 clinical development for the treatment of genetic disorders due to nonsense mutations. (cff.org)
Alligator Bioscience has announced an approved IND (Investigational new Drug) for the CD40 targeting antibody mitazalimab. (nordiclifescience.org)

Ginkgo Bioworks in a number of transactions, including a strategic partnership with Aldevron to optimize production of vaccinia capping enzymes (VCE), and a partnership with Berkeley Lights to use BLI's optofluidic platform in Ginkgo's genetic engineering foundries. (ropesgray.com)

What are best practices right now for data collection, ownership and sharing to benefit rare disease diagnosis, drug development and access. (terrapinn.com)
The rare disease space is a breeding ground for innovative approaches in drug development and platform technologies. (terrapinn.com)
Approaches in AI and digital health span the full development life cycle of drug development and commercialization. (terrapinn.com)
If you're looking to outsource drug product development to a CDMO, you need a collaborative partner who will engage you deeply to understand the unique needs of your project. (cambrextallinn.info)
Milestones in technological advancements in immunoglobulin production processes, such as development of new plasma purification methods and more effective antibodies, have offered robust backings to. (reportlinker.com)
Carol has about 20 years of drug development experience gained in small, medium and large pharma companies working in Germany, the UK and France. (xtalks.com)
The new agreement specifically involves the development and licensing of the drug in Greater China. (nordiclifescience.org)
The drug was safe and well tolerated, as judged by an independent safety committee, which "supports continued development," Ionis said in a news release . (medscape.com)

In addition to its proprietary precision diagnostics for oncology, Helomics offers boutique CRO services that leverage our patient-derived tumor models, coupled to a wide range of multi-omics assays (genomics, proteomics and biochemical), and a proprietary bioinformatics platform (D-CHIP) to provide a tailored solution to our client's specific needs. (globenewswire.com)

The foundation of the AOP Orphan subsidiary OrphaCare GmbH in 2018, with its focus on developing patient services, cemented this patient-centric approach. (aop-health.com)

Dr. Niihara is board-certified by the American Board of Internal Medicine/Medical Oncology and the American Board of Internal Medicine/Hematology. (emmausmedical.com)

The deal was based on the expected performance of Vertex's blockbuster new hepatitis C drug, Incivek. (binjonline.com)

The goal is to develop tailor-made AI solutions for Diamyd Medical's pharmaceutical production. (nordiclifescience.org)

New insight into a gene that controls energy production in cancer stem cells could help in the search for a more effective treatment for glioblastoma. (news-medical.net)

The new drug is, biologically speaking, more comprehensive than its statin predecessors: it inhibits the production of the protein PCSK9, which binds to the LDL but self-destructs when it enters the liver. (leadersleague.com)
For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated," Sarah Tabrizi, MD, PhD, director of the University College London Huntington's Disease Centre and global chief investigator of the phase 1/2a study of the drug, said in a statement issued by the university. (medscape.com)
Ionis-HTTRx is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant is responsible for Huntington's disease. (medscape.com)

KN026 is an anti-HER2 bispecific antibody invented by Alphamab Oncology using the proprietary Fc-based heterodimer bispecific platform technology called CRIB (Charge Repulsion Induced Bispecific). (weeklyreviewer.com)

So, within just the span of a couple of years, going from zero approved indication specifically for this rare skin condition to multiple agents, really shows the spectrum of what can happen in the orphan disease space. (atheneum.ai)
Rare disease pioneer AOP Orphan takes over two health care companies, strengthening its position in Europe as well as Vienna as a research- and business hub. (aop-health.com)

Physics and imaging in radiation oncology. (lu.se)

Cardiovascular disease is the world's number-one human-killer, and sales of drugs that combat it are expected to exceed $64 billion in five years' time. (leadersleague.com)

The dose-dependent reductions of mHTT we observed in the study substantially exceeded our expectations and we were equally encouraged by the safety profile of the drug," C. Frank Bennett, PhD, senior vice president of research at Ionis Pharmaceuticals said in the release. (medscape.com)

Our lead drug candidate, Berubicin, was developed by Dr. Waldemar Priebe, Professor of Medicinal Chemistry at The University of Texas MD Anderson Cancer Center. (cnspharma.com)

Metabolic enzymes (and the genes coding for them), which determine a drug s toxicity and side effects, could work so well that a drug is chopped up and excreted before it gets to work.So personalized medicine, if it has any chance of success, relies on treating genotypes rather than phenotypes, and having the means to test for the underlying characteristic. (pharmamanufacturing.com)

U.S. Food and Drug Administration (FDA). (wikipedia.org)
IND approval by the US Food and Drug Administration (FDA) is a prerequisite to start clinical trials in the USA. (nordiclifescience.org)

Asunercept has been granted orphan drug status for the treatment of GBM and MDS in the EU and the US. (wikipedia.org)
PTC124 has also been granted orphan drug status for the treatment of CF and DMD by the European Commission. (cff.org)
Mirdametinib, the company next most advanced drug candidate, is undergoing Phase 2b trial as a treatment for inoperable plexiform neurofibromas (NF1-PN). (cryptoculture.info)
Inappropriate use of second- and third-line regimens can contribute to drug resistance, limit treatment options and often have higher cost. (who.int)
We describe the recruitment and data collection challenges and recommendations for assessing DS in oncology treatment facilities. (cdc.gov)

We are happy to be working with recognized experts in both AI and sustainable production," says Ulf Hannelius, CEO of Diamyd Medical. (nordiclifescience.org)
Diamyd Medical, together with AI technology company MainlyAI and KTH (the Royal Institute of Technology), will collectively share SEK 13 million of funding from the Swedish Innovation Agency Vinnova for a project that will design, test and build a model for sustainable production using artificial intelligence. (nordiclifescience.org)

The orphan drug space is booming with innovation, M&A activity and partnerships. (terrapinn.com)
So the drug is an innovation - and will likely be very profitable. (leadersleague.com)

The AOP Orphan Group manufactures 80 percent of its drugs in Europe - an aspect which has become more and more relevant since the start of the COVID-19 pandemic. (aop-health.com)
The AOP Orphan Group now has more than 20 subsidiaries and representative offices across the whole of Europe as well as in the United Arab Emirates and Israel. (aop-health.com)
Our clinical drug supply of Berubicin is nearing completion via a redundant manufacturing strategy with production in the US and in Europe. (cnspharma.com)
The perceived imbalance between price and value for drugs has led to negative publicity for the industry in the US, market access delays in Europe and other industrialized countries, and suboptimal penetration in many markets. (pharmexec.com)

That drug is Inclisiran, a drug to treat familial hypercholesterolemia (a hereditary high-cholesterol disorder), which has recently passed through a series of Phase 3 clinical studies. (leadersleague.com)
The fact that levels of mutant huntingtin were reduced in correlation to the dose of Ionis-HTTRx that was given is significant, and the fact that participants in this first Phase 1/2a study are able to continue on the drug through open label extension gives us optimism regarding its safety," HDSA President and CEO Louise Vetter said in a statement. (medscape.com)

Among the innovative oncology drugs which appear on the 2012 draft list are: Novartis' Afinitor (everolimus), Eli Lilly's Alimta (pemetrexed), GlaxoSmithKline's Atriance (nelarabine), Merck Serono's Erbitux (cetuximab), AstraZeneca's Iressa (gefitinib), Bayer's Nexavar (sorafenib), Celgene's Revlimid (lenalidomide), Bristol-Myers Squibb's Sprycel (dasatinib), Pfizer's Sutent (sunitinib) and Novartis' Tasigna (nilotinib). (pharmatimes.com)

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Analytical, Diagnostic and Therapeutic Techniques and Equipment2

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Designation11

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American Society1

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Medicines4

AstraZeneca1

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