Tag: rare disease

CELEBRATION, Fla., July 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name MIPLYFFA®.

PARIS, FRANCE, 24 July 2025 - Ipsen announced today that the European Commission has approved Cabometyx^® (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues.

New Iso-Seq data from ~1,000 samples to expand transcriptomic insights using highly accurate HiFi sequencing

PARIS, FRANCE, 23 July 2025 - Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty care biopharmaceutical company, today announced the following changes to its Executive Committee:

NOVATO, Calif., July 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 45,292 restricted stock units of the company’s common stock to 29 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of July 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable

Daix (France), New York City (New York, United States), July 9, 2025 – Inventiva (Euronext Paris and Nasdaq: IVA) (“Inventiva” or the “Company”), a clinical-stage biopharmaceutical company focused on the development of oral therapies for the treatment of metabolic dysfunction-associated steatohepatitis (“MASH”), today announced a leadership transition with the appointment of Jason Campagna, MD, PhD, as President of Research and Development (“R&D”) and Chief Medical Officer (“CMO”) and Martine Zimmermann, PharmD, as Executive Vice President (“EVP”) of Regulatory Affairs and Quality Assurance.

Daix (France), New York City (New York, États-Unis), le 9 juillet 2025 – Inventiva (Euronext Paris et Nasdaq : IVA) (« Inventiva » ou la « Société »), société biopharmaceutique spécialisée dans le développement de thérapies orales pour le traitement de la stéatohépatite associée à un dysfonctionnement métabolique (« MASH »), a annoncé aujourd'hui une transition au sein de son équipe de direction avec la nomination de Jason Campagna, MD, PhD, en tant que Président de la Recherche et du Développement (« R&D ») et Directeur Médical (« CMO »), ainsi que Martine Zimmermann, PharmD, en tant que Vice-Présidente Exécutive (« EVP ») en charge des affaires réglementaires et de l’ assurance qualité.