Yesterday the agency denied approval for Abeona Therapeutics’ investigational gene-corrected cell therapy prademagene zamikeracel (pz-cel) for the treatment of recessive dystrophic epidermolysis bullosa, a rare disease that causes painful blistering and erosion of the skin.
CEO Hervé Hoppenot said on an investor call that Escient Pharmaceuticals’ two lead assets “address large populations with a clear medical need” with a potential multibillion-dollar market opportunity.
Data from the Phase III LUNA 3 study on Tuesday showed that Sanofi’s BTK inhibitor rilzabrutinib significantly improved durable platelet response in patients with chronic immune thrombocytopenia.
Nine months into the U.S. launch of the first drug proven to slow the advance of Alzheimer’s, Eisai and Biogen’s Leqembi is facing an unexpected hurdle to widespread use: an entrenched belief among some doctors that treating the memory-robbing disease is futile.
Pfizer and BioNTech asked a London court to revoke rival Moderna’s patents over technology key to the development of vaccines for COVID-19, as the latest leg of a global legal battle began on Tuesday.
Swiss drugmaker Novartis raised its full-year guidance on wider use of psoriasis and arthritis drug Cosentyx on Tuesday and said it had tapped the former boss of Bristol Myers Squibb to become its chairman next year.
The agency’s green light is a shot in the arm for ImmunityBio, which had reiterated doubts in a regulatory filing last month about its ability to remain in business.
Boehringer Ingelheim is paying $35 million in upfront and near-term fees to work with Ochre Bio to identify and validate regenerative targets for metabolic dysfunction-associated steatohepatitis and other chronic liver diseases.
In 2023, cell and gene therapy saw an unprecedented surge with seven FDA approvals, and this year, an even greater number of these treatments could reach the market. So far in 2024, the regulator has given the green light to three new CGTs, and at least seven additional cell and gene therapy products are expected to receive approval by year’s end, according to a March report from the Alliance for Regenerative Medicine.
The U.S. Supreme Court on Monday declined to hear a bid by Vanda Pharmaceuticals to revive patents for its sleep-disorder drug Hetlioz that were previously declared invalid in a dispute with generic drugmakers Teva and Apotex.
Reuters Health