April 28, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call today, April 28 at 4:30 p.m. ET to share results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam. PTC's partner Novartis stated on its quarterly earnings call today that long-term extension data are supportive of the now-initiated Novartis Phase 3 INVEST-HD study and Novartis and PTC will assess potential best next steps for the program including further actions wi

MENLO PARK, Calif.---Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy for the treatment of high-risk hematologic malignancies. "The FDA’s RMAT designation for Orca-Q recognizes the significant un

April 29, 2026 - Addex Therapeutics (SIX: ADXN and Nasdaq: ADXN), a clinical-stage biopharmaceutical company focused on developing a portfolio of novel small molecule allosteric modulators for neurological disorders, announced today encouraging preclinical data demonstrating antitussive activity of its GABAB positive allosteric modulator (PAM) candidate in a bleomycin (BLM)-induced idiopathic pulmonary fibrosis (IPF) exacerbated chronic cough model. “Chronic cough remains a significant unmet me

Clearance will accelerate development of a therapeutic for Complex Regional Pain Syndrome (CRPS) There are currently no approved treatment options for CRPS in the United States April 28, 2026 -- BRC Therapeutics (BRC), a pharmaceutical company developing therapeutics for neurological and inflammatory diseases, announced today that the company can proceed with a Phase I study to further establish safety of BRC-002 in healthy volunteers. BRC-002 is a first-in-class investigational product aiming

– If confirmed by the European Commission, REDEMPLO would be the first and only siRNA medicine authorized in the EU for both genetically confirmed and clinically diagnosed adult patients with FCS – People living with FCS have extremely high triglyceride levels and a substantially higher risk of acute pancreatitis and related long-term complications, often resulting in a reduced quality of life – The CHMP opinion is based on positive results from the Phase 3 PALISADE study where REDEMPLO reduced

April 24, 2026 -- Levicept Ltd, a biotechnology company focused on the development of LEVI-04, a first-in-class treatment for osteoarthritis (OA), will today present positive data from its Phase II study of LEVI-04 in patients with pain and disability due to OA of the knee at OARSI 2026 World Congress on Osteoarthritis being held in West Palm Beach, FL 23-26 April. Levicept has been selected for four presentations including a podium session in which Professor Philip Conaghan MBBS PhD, Director

Arrowhead Pharmaceuticals' therapy for familial chylomicronemia syndrome (FCS) appears to have gained a slight advantage over a competing drug from Swedish Orphan Biovitrum (Sobi) and Ionis Pharmaceuticals after an EMA panel backed approval in patients without the requirement for a genetic test. The positive opinion from the Committee for Medicinal Products for Human Use (CHMP) — disclosed on Friday — means that Arrowhead's small interfering RNA (siRNA) medicine Redemplo (plozasiran) can be use

Sanofi’s tolebrutinib is on track to get an approval in Europe for a type of multiple sclerosis even after receiving a complete response letter from the FDA in December. On Friday, the European Medicines Agency’s human medicines committee recommended approval of the BTK inhibitor for secondary progressive multiple sclerosis (SPMS) without relapses in the past two years. The decision was based in part on data from the Phase 3 HERCULES trial, which showed tolebrutinib delayed time to onset of six

U.S. Food and Drug Administration (FDA) issues Complete Response Letter (CRL) for trenibotulinumtoxinE (TrenibotE) related to manufacturing processes; no additional clinical studies are requested As the leader in neurotoxin development and manufacturing, AbbVie is well positioned to address all comments in a timely manner April 23, 2026 -- AbbVie (NYSE: ABBV) today announced that it received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the Biologic

April 22, 2026 -- Veloxis Pharmaceuticals, Inc. (Veloxis), a global specialty pharmaceutical company part of Asahi Kasei Corporation, announced today that pegrizeprument (also known as VEL-101), a novel investigational maintenance immunosuppressive agent, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the prophylaxis of heart allograft rejection in patients receiving a heart transplant. This follows the recent ODD granted for prevention of organ