EMERYVILLE, Calif. — XOMA Corporation the biotech royalty aggregator, announced today that based upon the U.S. Food and Drug Administration’s (FDA) acceptance of Zevra Therapeutics’ New Drug Application (NDA) for arimoclomol, an orally-delivered, first-in-class therapy for Niemann-Pick disease type C (NPC), XOMA will make a $1 million milestone payment to...
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Stockholm, Sweden – Sobi® (STO: SOBI), a global biopharmaceutical company dedicated to delivering innovative treatments for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) seeking approval for Nanoecapsulated Sirolimus plus Pegadricase (NASP), formerly SEL-212, for the treatment...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the company’s PALYNZIQ® (pegvaliase-pqpz) supplemental Biologics License Application (sBLA) to expand treatment to include adolescents aged 12-17 with phenylketonuria (PKU). The FDA has set a Prescription Drug User...
DEER PARK, Ill. — Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the Company’s New Drug Application (NDA) for ET-600, a proprietary patented formulation of desmopressin oral solution, has been accepted for review by...
PRINCETON, N.J. — Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage, biopharma company advancing GTx-104, a clinical-stage, novel, injectable formulation of nimodipine being developed for IV infusion to address significant unmet medical needs in aSAH patients, today announced that on August 22, 2025 the U.S. Food...
Regulatory submission is based on positive results from the ongoing Phase I/II SOHO-01 trial in patients with advanced HER2-mutant non-small cell lung cancer (NSCLC). Investigational agent sevabertinib (BAY 2927088) is an oral, small molecule, tyrosine kinase inhibitor (TKI) being evaluated as a potential new targeted therapy for patients with NSCLC...
Basel, Switzerland – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the US Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) for Gazyva®/Gazyvaro® (obinutuzumab) for the treatment of lupus nephritis. The filing acceptance is based on positive results from the phase III REGENCY study,...
On March 22, an FDA Advisory Committee (AdCom) unanimously voted in favor of accelerated approval for Biogen’s tofersen for a rare form of amyotrophic lateral sclerosis (ALS). Panelists voted 9-0 that tofersen’s demonstrated reduction in plasma neurofilament—a biomarker of neurodegeneration—was “reasonably likely” to predict clinical benefit in ALS. The AdCom...
Washington DC – The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee agreed that Vertex Pharmaceuticals had sufficiently demonstrated the safety of its investigational sickle cell disease gene-edited therapy exagamglogene autotemcel (exa-cel). Still, the panel of external experts did indicate that Vertex and its partner CRISPR Therapeutics could follow treated...
Most parents wouldn’t be thrilled with the idea of their kids getting hooked up to an IV bag filled with trillions of viruses. But for Melanie Hennick, whose son, Connor, has Duchenne muscular dystrophy, it was an opportunity she hoped would change his life. “We knew this wasn’t a cure,”...