Regenerative medicine company Mesoblast Ltd.’s stock sank nearly 57% on the news that it received a second U.S. FDA complete response letter (CRL) following the resubmission of its BLA for allogeneic stem cell treatment remestemcel-L in children with steroid-refractory acute graft-vs.-host disease. In the CRL, issued a few days after the Aug. 2 PDUFA date, the agency said it requires more data to support approval.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: 180, Aardvark, ABM, Orchard, Taiho.
The U.S. FDA recognized several new standards for sterilization of medical devices as part of an effort to reduce the use of ethylene oxide (EtO) for this purpose. While the recognition may stimulate adoption of alternative methods, Congress is applying yet more pressure on the EPA to act more decisively in regulating EtO, thus amplifying pressure on a system that device makers believe is already under stress.
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Irhythm Technologies, Quanta Dialysis Technologies.
Satellos Bioscience Inc. has received orphan drug designation and rare pediatric disease designation from the FDA for SAT-3153 for the potential treatment of Duchenne muscular dystrophy (DMD). The first-in-class oral small-molecule therapeutic is designed to restore the innate muscle regeneration process independent of dystrophin and regardless of exon mutation status.
While U.S. politics continues to delay Senate confirmation of the NIH director, other crucial positions at the agency that don’t require Senate action are being filled. Acting NIH Director Lawrence Tabak announced Aug. 2 that he has named Jeanne Marrazzo as director of the agency’s National Institute of Allergy and Infectious Diseases (NIAID).
While U.S. politics continues to delay Senate confirmation of the NIH director, other crucial positions at the agency that don’t require Senate action are being filled. Acting NIH Director Lawrence Tabak announced Aug. 2 that he has named Jeanne Marrazzo as director of the agency’s National Institute of Allergy and Infectious Diseases (NIAID).
The old adage of “what goes up must come down” rarely applies to U.S. FDA user fees. Most of the fiscal 2024 fees, which go into effect Oct. 1, continue in an upward slope. Under the PDUFA agreement, the fees for NDAs/BLAs are surging 25%. All but one of the MDUFA fees are going up 9.5%; the lone exception is growing by 18%. The MDUFA hikes are building on top of this year’s hefty increases. In the generics realm, the drug master file fee is going up 21%, but other fees increases are in the single digit range. Biosimilars, however, are the exception to the rule, with all the BsUFA fees seeing big drops.
The European Union and the U.S. have wrapped up a data privacy framework that covers broad swaths of both economies, including the transmission of clinical trial data across the Atlantic Ocean. Drug and device makers that want to make use of this framework and thus jettison the contractual clause to ensure data privacy may find compliance with this new framework much more efficient in the long run, but will have to do a lot of compliance work on the front end to achieve those efficiencies.
Premature babies face challenges in the neonatal intensive care unit (NICU) as their heart, lungs and neurological system continue to develop outside the womb. Roughly half of neonates born at 26 weeks to 28 weeks gestation experience respiratory distress. These infants often need extra oxygen and help breathing, as well as constant monitoring to ensure oxygen is absorbed and carbon dioxide (CO2) is dispelled. To that end, Etiometry Inc. received FDA clearance for its IVCO2 Index, a software tool for use in monitoring risk of hypercapnia in NICU patients weighing less than 2 kg.
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