Rare Diseases

Ovid Therapeutics' president and COO Meg Alexander is leading the company’s strategic pivot toward innovative neurological treatments, potentially creating a new class of medicines for rare neurological disorders.

BioMarin’s chief business officer James Sabry told Scrip that Inozyme’s enzyme replacement therapy INZ-701 aligns with the company’s existing portfolio and its areas of dealmaking focus.

BioMarin’s ultra-rare disease drug Brineura has breached cost-effectiveness thresholds for highly specialized treatments in England and looks set to be denied routine funding on the National Health Service.

Sarepta’s stock price was battered after Vinay Prasad was appointed to lead the US FDA’s CBER, then the company’s shares were bruised when Q1 Elevidys sales came in below consensus.

Vertex is in need of diversification, but in addition to its lower-than-expected Q1 revenue, sales of the company’s newest CF drug Alyftrek and pain medicine Journavx disappointed.

EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.

BridgeBio reported $36.7m in Q1 2025 sales of its ATTR-CM drug Attruby, tripling consensus forecasts. Vyndaquel-maker Pfizer acknowledged the product’s impact during the quarter.

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

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 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.

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.

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.