Receiving a second complete response letter (CRL) from the U.S. FDA for gefapixant to treat refractory and unexplained chronic cough – an indication for which there are no approved treatments in the U.S., Merck & Co. Inc. said it is reviewing the agency’s feedback to determine the next steps, if any, for the oral selective P2X3 receptor antagonist.
A threat to biopharma innovation arrived at a larger scale in 2023 in the form of greater U.S. FTC scrutiny, calling into question the legitimacy of certain M&As and deals.
Putting into jeopardy what was on track to be the first approved therapy in Europe for geographic atrophy (GA), an advanced form of age-related macular degeneration that causes blindness, Apellis Pharmaceuticals Inc. learned of a negative trend vote by the EMA’s Committee for Medicinal Products for Human Use (CHMP) on its MAA for intravitreal pegcetacoplan.
Sanofi SA backed out of its $750 million effort to advance Maze Therapeutics Inc.’s oral Pompe disease candidate, MZE-001, after the U.S. FTC filed a federal lawsuit to block the deal, claiming the Paris-based firm was seeking to eliminate a nascent competitor.
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
The oral drug inavolisib, when added to two other therapies, significantly improved progression-free survival in the first-line phase III treatment of advanced hormone receptor-positive, HER2-negative breast cancer in which patients have a PIK3CA mutation.
Redhill Biopharma Ltd.’s stock (NASDAQ:RDHL) has soared in recent days following news that the U.S. FDA granted five years of market exclusivity for Talicia, a first-line therapy for eradication of Helicobacter pylori infection, which affects about 35% of the U.S. adult population.
Eli Lilly and Co., through its Loxo@Lilly oncology unit, secured its second accelerated approval for non-covalent Bruton’s tyrosine kinase (BTK) inhibitor Jaypirca (pirtobrutinib), this time to treat adults with chronic lymphocytic leukemia or small lymphocytic lymphoma. The U.S. FDA approval of 100-mg and 50-mg tablets is for patients who have received two prior lines of therapy, including another BTK inhibitor and a BCL-2 inhibitor. It is based on phase I/II data from a subset of 108 patients participating in the open-label, single-arm, multi-cohort Bruin trial.
Redhill Biopharma Ltd.’s stock (NASDAQ:RDHL) has soared in recent days following news that the U.S. FDA granted five years of market exclusivity for Talicia, a first-line therapy for eradication of Helicobacter pylori infection, which affects about 35% of the U.S. adult population.
The U.S. FDA put out a safety warning Nov. 28 that antiseizure drugs levetiracetam and clobazam can cause a rare but serious hypersensitivity drug reaction that may start as a rash but can progress to injure internal organs. In addition, U.K. health care providers are being told to get a plan in place now to implement the first phase of the Medicines and Healthcare products Regulatory Agency’s new regulatory measures to reduce the risks of valproate, a treatment for epilepsy and bipolar disorder.
