Neuromuscular disease specialist NMD Pharma A/S has been given U.S. FDA approval for a phase IIb trial of NMD-670, after demonstrating proof of mechanism for the orally-available chloride ion channel-1 inhibitor in generalized myasthenia gravis.
The U.S. FDA has posted another proposed ban of the use of electrical stimulation devices (ESDs) for the treatment of self-injurious behavior, which revisits the controversy over their use by the Judge Rotenberg Center.
Kazia Therapeutics Ltd. has out-licensed paxalisib as a potential treatment for intractable epilepsy in focal cortical dysplasia type 2 and tuberous sclerosis complex disease in a carve-out deal with Sovargen Co. Ltd. for $20.5 million plus sales royalties.
In February, the U.S. FDA greenlit 11 new drugs, a slight decrease from the 14 approvals seen in the same month the previous year but an increase from January’s tally of eight FDA approvals.
Stimlabs LLC received U.S. FDA approval for Corplex P, a wound care product that uses placental tissue to facilitate wound healing. “Corplex P is the first particulate device derived from human umbilical cord extracellular matrix indicated for the management of acute and chronic wounds,” Stimlabs CEO John Daniel told BioWorld.
The U.S. FDA has approved Duvyzat (givinostat), from Italfarmaco SpA, for treating Duchenne muscular dystrophy (DMD). It is the first oral, nonsteroidal drug for treating all of DMD’s genetic variants. The oral treatment is approved for those ages 6 and older.
Vertex Pharmaceuticals Inc. has obtained IND clearance from the FDA for VX-407, an investigational first-in-class small-molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
Triarm Therapeutics Ltd. is on a mission to democratize CAR T therapies to make them more accessible and affordable, Triarm CEO Jay Zhang told BioWorld. “Nearly half of the patients eligible for CAR T therapies still cannot get treated, and the main reason is the expense, and the second is they cannot afford to wait.
The U.S. FDA’s pending final rule for regulation of lab-developed tests has proven unusually controversial even for the FDA, but Rep. Anna Eshoo (D-Calif.) stated in a March 21 hearing that congressional inaction has left the agency in an uncomfortable spot.
As part of its required series of guidances on using real-world evidence, the U.S. FDA released a draft guidance in response to sponsors’ growing interest in the potential use of observational studies to contribute to a demonstration of the effectiveness or safety of a drug or biologic.
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