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Rare Diseases

SpringWorks To Make Its Case For Nirogacestat At High-Stakes EMA Meeting

Nirogacestat was one of four products scheduled for an oral explanation meeting at the European Medicines Agency this week.

EMA To Consider Approvals For Eight Orphans, Including Vertex’s CF Drug Alyftrek

The European Medicines Agency is this week expected issue opinions on whether the European Commission should grant EU marketing authorization for 19 new products.

Roivant Tightens Leadership Grip On Immunovant, Narrows Near-Term IMVT-1402 Focus

Roivant president Eric Venker was named CEO of Immunovant to guarantee execution of the development plan for IMVT-1402 in six indications, now including Sjögren’s disease and cutaneous lupus erythematosus.

Merck’s Digital Biomarker Strategy For Commercial Gains In Neurology

The integration of digital health tools into clinical research and patient care is gaining momentum, and Merck KGaA, Darmstadt, Germany is not letting the grass grow under its feet. With an approach to digital biomarkers, the company is seeking to redefine how neurological diseases are monitored and managed.

Podcast: iOnctura’s Mission To Treat Rare Cancers With Precision Therapies

In Vivo spoke with Catherine Pickering, the CEO and founder of iOnctura, a UK-based clinical stage biotech company developing therapies for uveal melanoma and other hard-to-treat cancers by disrupting the tumor-stroma-immune interface.

Samsung Rolls Out Second US Soliris Biosimilar – At 30% WAC Discount

Samsung Bioepis and Teva have provided clarity on the pricing of their Epysqli biosimilar to Soliris in the US, as the firms launched only the second rival to the rare diseases treatment.

Amgen’s Rare Disease Portfolio Grows With Second Approved Uplizna Indication

The US FDA approved anti-CD19 antibody Uplizna, from Amgen’s $27.8bn purchase of Horizon in 2023, for IgG4-related disease – a larger market than its original NMOSD indication.

Sponsors Of Two Orphans And An Acne Treatment To Defend Marketing Authorization Applications At EMA

Sponsors of three drugs that are in the final stages of the EU regulatory review cycle are due to make the case for marketing approval before the European Medicines Agency.

Regenxbio’s Triple Threat: Partnered Programs, DMD Progress And In-House Manufacturing

Regenxbio is riding a wave these days, with all three of its clinical-stage programs set to reach critical milestones this year and recent positive news on its DMD gene therapy, just as Sarepta suffered a setback with a death related to its DMD treatment. In Vivo spoke with CEO Curran Simpson about the company’s plans and strategic decisions.

Mirum’s Rare Disease Strategy: A Blueprint For Sustainable Growth

From a $7m discarded asset to a $336m commercial portfolio, In Vivo looks inside Mirum Pharma’s disciplined strategy for identifying, acquiring and revitalizing overlooked rare disease programs while building toward billion-dollar potential.