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Ralph Abraham, a vocal vaccine skeptic who served at the agency for just three months, has stepped down due to “unforeseen family obligations,” according to the CDC.
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The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra.
Maintaining America’s momentum demands that policymakers resist policies that undermine research and development incentives.
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Read our takes on the biggest stories happening in the industry.
Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.
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The combined company began trading Friday under the Nasdaq symbol TECX. A $130 million private placement was also completed, with a cash runway into mid-2027.
Jazz Pharmaceuticals’ calcium channel modulator suvecaltamide in a mid-stage trial was unable to significantly lower tremor burden in patients with essential tremor, the company reported Thursday.
Vertex Pharmaceuticals’ three cystic fibrosis drugs—Kaftrio/Trikafta, Symkevi and Orkambi—will now be available through England’s National Health Service, following the signing of a long-term reimbursement agreement.
Vanda Pharmaceuticals has rejected two unsolicited takeover offers, saying that they are “opportunistic attempts” to acquire the biotech at a heavily discounted price.
While Thursday’s label expansion and traditional approval for the gene therapy is an important milestone, many challenges still face the Duchenne muscular dystrophy community.
Zealand Pharma’s petrelintide cut body weight by more than 8% on average, with a good overall safety and tolerability profile.
On the heels of a Phase III flop for Pfizer’s Duchenne muscular dystrophy gene therapy candidate, the FDA has green lighted the expanded use of Sarepta Therapeutics’ Elevidys.
The pharma industry is staring down the barrel of a widespread loss of exclusivity, with more than 190 products going off-patent between 2022 and 2030. Here are some strategies company are employing to manage the drop in revenue.
Gilead Sciences reported zero cases of HIV infection occurred in the lenacapavir candidate group, compared to 16 cases in the Truvada arm and 39 incidents for those treated with Descovy, Gilead’s approved daily PrEP pills.
PTC Therapeutics said Thursday the FDA has lifted a partial clinical hold on its Huntington’s disease candidate PTC518 after displaying favorable clinical trends in a mid-stage study.