FDA
The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
FEATURED STORIES
The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
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Please check out the biopharma industry’s COVID-19 stories that are trending for May 4, 2021.
Sarepta Therapeutics announced Monday that its next-generation PPMO candidate, SRP-5051, showed promise in a Phase II trial when used as a treatment for Duchenne muscular dystrophy in patients amenable to exon 51 skipping.
The U.S. FDA is expected to give the go-ahead for Pfizer and BioNTech’s COVID-19 vaccine for adolescents ages 12 and older within the week.
The U.S. FDA approved Chiesi Global Rare Diseases’ Ferriprox for treatment of transfusional iron overload caused by sickle cell disease (SCD) or other anemias in adults and children ages three years and older. This approval expands the use of the drug for patients with SCD or other anemias.
Roivant Sciences is combining with Montes Archimedes Acquisition Corp (MAAC). Once the deal is closed, outstanding shares and warrants of MAAC will be traded for newly issued shares and warrants of the company.
The regulatory nod marks the first time the FDA approved an SGLT2 inhibitor for the treatment of CKD regardless of diabetes status.
The U.S. FDA has released a Complete Response Letter (CRL) following its review of LEO Pharma’s tralokinumab. Further data on a device component is the issue brought to light by the recently completed Biologics License Application (BLA) review.
The U.S. FDA says it is unable to approve Biogen supplemental Biologic License Application (sBLA) for subcutaneously delivered TYSABRI® for the treatment of relapsing multiple sclerosis (MS), at least while the sBLA remains in its present form.
The FDA is threatening Acceleron with a $10,000 fine or criminal prosecution, charging the company with failure to post a summary of study data on its cancer combo dalantercept and axitinib in the ClinicalTrials.gov online database.
Moderna, one of three companies to have a COVID-19 vaccine authorized in the U.S., isn’t resting on its accomplishments but is instead working to scale up production further and improve on its vaccine.