FDA
Disc Medicine’s leadership tried to express optimism that its rare disease therapy bitopertin can be approved based on a Phase 3 trial set to begin shortly. However, analysts are worried that the protocol was developed with former FDA leaders.
FEATURED STORIES
The FDA issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
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.
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The companies on Wednesday secured the regulator’s approval for their eye drop Ryzvumi for the reversal of pharmacologically-induced mydriasis, as Viatris looks to add $1 billion in sales by 2028.
The FDA on Monday approved the Canadian biopharma’s liquid antibiotic metronidazole, an alternative for patients who are unable to use pills or injections.
ARS Pharmaceuticals, Intarcia Therapeutics and Taysha Gene Therapies this week got stark reminders of the difficulties in getting treatments through the regulator’s approval process.
Following a regulatory victory for Jemperli in endometrial cancer last month, GSK continued its oncology winning streak with an FDA approval for momelotinib in adult myelofibrosis patients with anemia.
The FDA will finish September with three action dates and one of the year’s most highly-anticipated advisory committee meetings.
The regulator Monday approved the companies’ supplemental Biologics License Applications for their respective mRNA shots formulated to more closely target currently circulating variants.
Amid a supply shortage of the attention-deficit/hyperactivity disorder and binge-eating disorder medication, the regulator has cleared a slew of generic drugs of Takeda’s Vyvanse pill.
However, a lack of regulatory harmonization risks undermining the effective implementation of these technology-driven approaches.
Bristol Myers Squibb announced Monday that Reblozyl is now approved as a first-line treatment for adults with anemia due to myelodysplastic syndromes who may require regular blood transfusions.
A second trial shows Novo Nordisk’s Wegovy improves heart health. Meanwhile, many other drugs are beginning to face generic competition, including from newly approved biosimilars.