The ultra-rare skin disease X-linked hypohidrotic ectodermal dysplasia (XLHED) can be a heavy burden for children, affecting structures in the skin including sweat glands, respiratory glands, and hair as well as teeth. Though lacking a specific treatment to date, a pivotal clinical trial planned by the Geneva-based nonprofit Esperare Foundation and Pierre Fabre SA could now help bring one about.
Blueprint Medicines Corp.’s cancer drug Ayvakyt (avapritinib) looks set to gain an expanded label in Europe, amid a flurry of decisions from the European Medicines Agency’s CHMP scientific committee. Late last week the CHMP gave a positive opinion for Ayvakyt for treatment of adults with advanced systemic mastocytosis, meaning the drug is likely to gain a further European indication in the coming weeks.
Mighty Libtayo has stumbled. Because Regeneron Pharmaceuticals Inc. and Sanofi SA couldn’t find common ground with the FDA on postmarketing studies, the two are voluntarily withdrawing the sBLA for Libtayo (cemiplimab-rwlc) as a second-line treatment for advanced cervical cancer. Discussion about the matter continues outside the U.S., the companies said.
Bristol Myers Squibb Co.’s Breyanzi (lisocabtagene maraleucel) CAR T-cell therapy has passed muster with European regulators as a therapy for relapsed or refractory large B-cell lymphoma, setting up a likely European marketing authorization in the coming weeks.
Several pharma companies think that targeting a rogue protein known as alpha-synuclein could be the key to halting or reversing neurodegeneration in Parkinson’s disease – but has the industry learned from a string of failures in Alzheimer’s? In the space of a few weeks, Novartis AG and Sanofi AS have signed major deals for molecules targeting misfolded alpha-synuclein, the rogue protein thought to be the root cause of Parkinson’s disease.
Pfizer Inc.’s oral antiviral Paxlovid (nirmatrelvir) COVID-19 antiviral looks set for approval in the European Union after it was given the green light by its top regulatory committee. The EMA’s Committee for Medicinal Products for Human Use recommended conditional marketing authorization for treatment of COVID-19 in adults who don’t require supplemental oxygen but are at increased risk for progressing to severe disease.
Touting a series of firsts and a premium price tag to match, Immunocore Holdings plc is poised to launch the uveal melanoma drug tebentafusp in the U.S. following FDA approval for the medicine. The regulatory nod makes the drug, branded Kimmtrak, the first T-cell receptor-based therapy to reach the market, the first approval for a drug targeting gp100, and the first drug approved in 40 years for the cancer, which is the most common eye cancer in adults, though still rare.
Leyden Laboratories BV has raised $140 million in a series B financing round to develop drugs that treat whole families of viruses, including a wide-spectrum flu antibody licensed from Johnson & Johnson’s Janssen Pharmaceuticals Inc. unit earlier this month.
Merck & Co Inc. has had a setback with its chronic cough drug gefapixant after the FDA rejected its NDA, a decision that also briefly hit the share price of rival Bellus Health Inc. before it regained market traction late Jan. 24. The FDA had been reviewing gefapixant since March 2021, but the regulator is now asking for additional information related to measurement of efficacy in a dreaded complete response letter. Merck said the response was not related to the safety of gefapixant, a P2X3 receptor antagonist, under development for treatment of refractory chronic cough (RCC) or unexplained chronic cough in adults. Shares in Merck (NYSE:MRK) ticked down 1.4% to $78.86 while Bellus Health shares (NASDAQ:BLU) rose by 1.8% to $5.66.
Administering multiple booster doses against COVID-19 is not sustainable in the longer term, international drug regulators said during a meeting of the International Coalition of Medicines Regulatory Authorities, held Jan. 12. Regulators argued that a long-term strategy should involve vaccines capable of tackling several variants at once, not just omicron, and that tweaking existing vaccines and comparing them with first-generation vaccines using clinical studies is the way forward, according to a report from the meeting.
