Emerging Therapies for Niemann-Pick Disease Typ...

UCB announces FDA approval of ZILBRYSQ for the Treatment of Adults with Generalized Myasthenia Gravis On the 17th of October 2023, UCB (Euronext Brussels: UCB) made an announcement regarding the approval of ZILBRYSQ® (zilucoplan) by the US FDA for the management of generalized myasthenia gravis (gMG) in adult pa...

Ardelyx has struck gold on its third attempt with Xphozah (tenapanor), the chronic kidney disease medication. Following two prior rejections, the FDA has granted its long-awaited approval. Xphozah, an innovative phosphate absorption inhibitor, is now officially sanctioned for the management of serum phosphate level...

Bristol Myers Squibb’s Zeposia has lost its exclusive status as the sole S1P receptor modulator available for ulcerative colitis treatment. The FDA has granted approval for Pfizer’s ulcerative colitis medication, Etrasimod, under the brand name Velsipity. This decision comes after the publication of Phase III resul...

South Rampart Pharma Receives U.S. FDA Fast Track Designation for SRP-001 for Acute Pain On October 12, 2023, South Rampart Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) had granted Fast Track status to its drug candidate, SRP-001, intended for the management of acute pain. SRP-001 is a...

Rare diseases affect a larger population than cancer and AIDS combined, with over 7,000 distinct conditions impacting more than 300 million globally. Approximately 1 in 10 individuals are affected by a rare disease, and it often requires several years to receive an accurate diagnosis, averaging around 4.8 years. Ap...

Fabre-Kramer Pharmaceuticals’ major depressive disorder drug, gepirone, faced FDA rejection not once, not twice, but three times since the beginning of the 21st century. Finally, the company can rejoice as the FDA has granted long-awaited approval. On 29 September 2023, the FDA approved Fabre-Kramer Pharmaceuticals...