rare disease
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MedCity Pivot Podcast: Tackling Rare Disease With Someone Who Was Touched By It
Rich Horgan believes that there’s an alternative path to drug development that can lower costs and the time it takes to bring drugs to market. He is trying to apply it to the field of rare and ultra-rare diseases.
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Biogen Bulks Up in Rare Disease With $7.3B Reata Pharmaceuticals Acquisition
Biogen’s Reata Pharmaceuticals acquisition brings Skyclarys, the first and only FDA-approved therapy for the rare neuromuscular disease Friedreich’s ataxia. Biogen says Skyclarys complements the other neuromuscular drugs in its portfolio.
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Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
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FDA Refuses to Review Biohaven Drug for Rare Neuromuscular Disease With No Treatments
Biohaven’s bid to bring to market the first drug for rare neuromuscular disorder spinocerebellar ataxia has hit a setback. The FDA is refusing to review the drug’s application, a decision that could indicate the agency wants another clinical trial.
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Here’s Why Novartis Is Paying $500M to Acquire a Preclinical RNA Startup
Novartis’s acquisition of DTx Pharma brings a lead program for a rare disease with no FDA-approved therapies. But the deal could also help the pharma giant bring new RNA therapies to new places in the body, a top Novartis executive explained.
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RA Capital Leads $150M Financing for Septerna and Lead Drug With Oral Edge in Rare Hormone Disease
Septerna Therapeutics’ Series B financing will support plans to reach the clinic with a pill for a rare disease whose only approved therapy is a Takeda Pharmaceutical drug that’s leaving the market. Potential rival therapies are all injectables.
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UCB Muscle Disease Drug Wins FDA Nod on Heels of Rival’s Regulatory Approval
The FDA approved UCB’s Rystiggo for treating generalized myasthenia gravis. Drugs are already available for the rare neuromuscular disorder, but Rystiggo’s approval includes treatment of a subset of patients not addressed by other products.
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Ipsen Itching Drug Scratches Off a Second FDA Approval in Rare Liver Indication
Ipsen drug Bylvay is now FDA approved for treating pruritus, or severe itching, which is a complication of the rare liver disease Alagille syndrome. The oral drug was previously approved for treating pruritus in another rare inherited liver disease called PFIC.
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Novartis Lines Up $3.2B Deal for Kidney Disease Drugs, But FTC Questions Loom
Novartis is acquiring Chinook Therapeutics in a $3.2 billion deal that brings two late-stage drug candidates for a rare kidney disorder. The Novartis drug pipeline already has a molecule in late-stage development for the same kidney disease, which could raise questions from antitrust regulators.
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FDA Approves First Drug for Rare Skin Disorder, and It’s a Redosable Gene Therapy
Krystal Biotech’s Vyjuvek is approved to treat recessive and dominant forms of dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. The redosable gene therapy, applied to the skin, delivers a gene that gets cells to produce collagen that DEB patients lack.
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Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.
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Rare Disease Drug Approval Brings New Competition to Meds from Sanofi, Takeda
A drug developed by Protalix BioTherapeutics and Chiesi Group is now FDA approved for treating Fabry disease, a rare inherited metabolic disorder. The drug, Elfabrio, is an enzyme replacement therapy.
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FDA Approval of Travere Kidney Disease Drug Sets Up Blockbuster Expectations
Travere Therapeutics drug Filspari won accelerated FDA approval to treat a rare disorder that can lead to kidney failure. The small molecule is also in late-stage testing for another rare kidney disease; analysts say each indication represents a blockbuster opportunity.
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Drug Approvals in 2022: A Recap of Notable FDA Regulatory Decisions
Fewer new drugs won FDA approvals in 2022 compared to previous years, but some of the products that passed regulatory muster are indicative of trends expected to continue into 2023. For example, more gene and cell therapies are on track to face regulatory decisions in the coming year.
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Vera’s Drug for Rare Kidney Disease Hits Trial Goals. Can It Stand Out in the Field?
Atacicept, a Vera Therapeutics drug in development for the rare kidney disease immunoglobulin A nephropathy, is on its way to a pivotal test after achieving the main goal of a mid-stage clinical trial. But the results still fall short of data recently reported by a competitor that is addressing the same disease target.
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How a pediatric hospital’s rare disease sequencing system is unlocking the whole genome
Children’s Mercy is claiming to build “the most advanced genomic sequencing system in the world” through its novel use of 5-base sequencing. Since launching three years ago, the system has already provided 1,000 children with rare disease diagnoses based on its genome sequencing.
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Acelyrin’s $300M haul for novel biologic leads a slate of biotech financings
The biggest biotech financing of the past week belongs to a startup developing an immunology drug that could rival blockbuster products sold by big pharmaceutical companies. Cell therapy, cancer, and rare disease also figured into this week’s startup cash hauls.
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Payer’s Place: Dr. Anil Singh
Dr. Anil Singh shares his insights into the strategies employed by the organization to identify the most effective digital solutions for their members.
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Discover the Next-Gen Platform for Integrated Collaborative Care
Beyond EHRs and digital front doors, reducing the gaps in patient care journeys.
