While some states are beginning to double down on the prices they pay for prescription drugs, the state of Colorado is taking it to a whole new level with its Prescription Drug Affordability Review Board that was empowered to set maximum prices of prescription drugs it considers “unaffordable.”
Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment, PRAX-628, resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%.
Regenerative medicine company Mesoblast Ltd. saw its stock shoot up 45% on the news that the U.S. FDA is satisfied with the additional data submitted from the company’s phase III study for remestemcel-L for treatment of adults with steroid-refractory acute graft-vs.-host disease (SR-aGVHD) to support filing a BLA in pediatric patients with SR-aGVHD.
Nuvation Bio Inc. said on March 25 it will acquire Anheart Therapeutics Ltd., a U.S.- and China-based precision oncology company, in an all-stock transaction, adding Anheart’s ROS1-inhibiting lung cancer drug, taletrectinib (AB-106), to its pipeline.
The U.S. FDA granted emergency use authorization (EUA) on March 22 for Waltham, Mass.-based Invivyd Inc.’s half-life extended monoclonal antibody (MAb) VYD-222 – making it available to prevent COVID-19 in immunocompromised adults and adolescents – but its use comes with a boxed warning for anaphylaxis.
The week began with PDUFA-delaying news for Regeneron Pharmaceuticals Inc. The company said it received two complete response letters (CRLs) from the U.S. FDA regarding the priority BLA for its bispecific antibody to treat lymphoma, odronextamab.
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.
Following the U.S. FDA’s March 11 approval of Novo Nordisk A/S’ glucagon-like peptide 1 receptor agonist, Wegovy (semaglutide), to reduce the risk of major adverse cardiovascular events such as heart attack and stroke, the Centers for Medicare & Medicaid Services (CMS) has issued guidance ensuring coverage.
When it comes to whether Medicare Part D should cover the new anti-obesity drugs, the U.S. Centers for Medicare & Medicaid Services and lawmakers may be caught between the math and public pressure.
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.