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A decade-long journey has come to an end for Stealth BioTherapeutics and the Barth syndrome community with the first-ever treatment for this uncommon mitochondrial disease. CEO Reenie McCarthy called it a “pivotal victory” that “offers hope for expedited regulatory attention to other ultra-rare diseases.”
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Writing in JAMA, four former government officials warn that the Trump administration’s involvement in delaying the approval of Novavax’s COVID-19 vaccine could indicate a politicization of the drug approval processes that could ‘imperil public health.’
Leading companies spent $1.4 billion upfront on licensing deals and embarked on vast R&D programs. Clinical setbacks mean many companies are unlikely to ever recoup their investments.
With much to cover, Democrats tackled Kennedy’s MAHA report; the firing of all members of the CDC’s ACIP committee; and much more. Little was accomplished, as Kennedy demurred and members of Congress accused him of risking American lives.
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AbbVie (NYSE: ABBV) today announced positive top-line results from SELECT-GCA, a Phase 3, multicenter, randomized, double-blind, placebo-controlled study, showing upadacitinib (RINVOQ®; 15 mg, once daily) in combination with a 26-week steroid taper regimen achieved its primary endpoint of sustained remissiona from week 12 through week 52 in adults with giant cell arteritis (GCA).
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Unpredictable communication and a lack of transparency are eroding the industry’s and the public’s trust. The FDA, experts agree, needs to take control of the narrative.
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Hundreds of HHS Staffers Accuse RFK Jr. of ‘Sowing Public Mistrust’ Against CDC After Shooting at HQ
In an open letter, Health and Human Services employees asked the Secretary to stop and disavow the spread of health misinformation, particularly about vaccines, infectious diseases and federal health agencies.
The delays, first revealed in Regeneron’s Q2 report, were chalked up to manufacturing issues at Novo Nordisk-owned plants.
Looking for a biopharma job in Massachusetts? Check out the BioSpace list of nine companies hiring life sciences professionals like you.
In late May, a patient died after receiving Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, spurring the hold. After lowering the dose and changing the regimen of immune modulators patients receive, the company has received FDA clearance for the trial to continue.
The FDA recommends that companies use overall survival as a primary endpoint for clinical trials where feasible. The new guidance follows the surprising return of CBER Head Vinay Prasad, who has previously argued for prioritizing OS.
With its structural changes, CSL expects to generate $500 to $550 million in annualized savings over the next three years.
Regulations aiming to lower the cost of vital medicines will instead end up restricting access and disincentivizing R&D.
The CDC no longer recommends COVID-19 vaccines for healthy children and healthy pregnant women, a position that has been opposed by leading medical societies.
A draft copy of an upcoming MAHA report reveals a strategy in lockstep with recent HHS actions such as reviving the Task Force on Safer Childhood Vaccines; Viking Therapeutics reports robust efficacy from mid-stage oral obesity candidate but is tripped up by tolerability concerns; Novo Nordisk wins approval for Wegovy in MASH; and Lilly takes a pricing stand.
Media coverage can help biopharma executives connect with, inform and inspire the public. In this column, Kaye/Bassman’s Michael Pietrack and three communications experts share how to make the most of these opportunities.