Food and Drug Administration (FDA)
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Fulcrum achieved what the company is calling a clinical first. Data showed that using losmapimod slowed the progression of FSHD and demonstrated improved function in patients.
The drug was approved on June 7 under the agency’s accelerated approval pathway, despite the agency’s Peripheral and Central Nervous System Drugs Advisory Committee voting against the drug in November 2020 and having been assured at that time the agency was not considering an accelerated approval.
The medical regulator’s green light is a milestone in the global fight against specific types of late-stage blood cancer, as CAR T-cell therapy has been found to be effective against them.
Cantex forges a licensing deal with vTv Therapeutics and intends to evaluate the vTv drug as a potential treatment for several cancer-related issues.
By merging with New Haven’s SPAC Thimble Point Acquisition Corp., Pear will be valued at $1.6 billion.
The U.S. CDC issued new guidance for long COVID, and the NIH is figuring out how to deal with the disease, while Tonix Pharma plans to develop TNX-102 SL.
Incyte had a particularly big week scheduled with the U.S. FDA, which included two PDUFA dates and an advisory committee meeting. In addition, Ascendis had a target action date.
The FDA said that arimoclomol, which has been studied and submitted for NPC indication, needs further data to support its use in this rare, progressive genetic disorder.
New data shows Zolgensma, Novartis’ gene therapy for spinal muscular atrophy, has the potential to be used presymptomatically in juveniles.
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