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Sarepta, Arrowhead take a big swing on siRNA drugs for rare disease

Sarepta Therapeutics and Arrowhead Pharmaceuticals teamed up on an siRNA partnership that has one of the biggest promises of biobucks in recent years. The deal, announced Tuesday morning, will see...

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Axsome moves toward NDA filing for narcolepsy drug after Phase 3 data

Axsome Therapeutics’ narcolepsy drug AXS-12 hit the primary endpoint in its Phase 3 ENCORE trial, showing a statistically significant improvement in the frequency of cataplexy attacks in narcoleptic...

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The Biden administration wants Medicare and Medicaid to cover GLP-1s. Will...

The Biden administration proposed a new rule Tuesday that would expand Medicare and Medicaid coverage of popular obesity drugs, earning praise from the pharmaceutical industry and patient advocates....

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Trump picks Stanford academic and pandemic critic Bhattacharya for NIH

President-elect Donald Trump tapped two policy and political outsiders to fill the remaining top healthcare roles in his administration, including Jay Bhattacharya, a prolific critic of the US Covid-19...

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Idorsia eyes layoffs, tees up Tryvio licensing deal to extend cash runway

Idorsia is making moves to stretch its cash reserves further. The Swiss drugmaker said it is narrowing its operations with 270 positions at risk of elimination. The potential layoffs will most likely...

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Acadia snaps up an essential tremor drug after field's summer setbacks

A few weeks after gaining about $100 million from a priority review voucher, Acadia Pharmaceuticals is putting its money to work by in-licensing an experimental essential tremor medicine. The San Diego...

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PTC reports Phase 2 ALS fail, sells priority review voucher for $150M

PTC Therapeutics is staying busy ahead of the Thanksgiving holiday. On Tuesday afternoon, the company announced that a Phase 2 trial evaluating its experimental ALS drug utreloxastat failed on the...

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Norbert Bischofberger's time as Kronos CEO ends as biotech seeks new life

Kronos Bio is letting go nearly all its employees after flashing red lights earlier this month. As part of an 83% workforce downsizing, CEO Norbert Bischofberger is heading out the door and handing the...

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Bristol Myers sues the federal government over 340B, following other drugmakers

Bristol Myers Squibb is the latest pharma company to sue the federal government over its rejection of a proposed new model for distributing 340B drug discounts. Under the drugmaker's plan, hospitals...

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Recursion reduces workforce; Oxford Nanopore teams up with UK Biobank

Plus, news about Portage Biotech: Recursion’s layoffs: After merging with fellow AI-driven biotech Exscientia this fall, Recursion Pharmaceuticals laid off less than 20% of the combined company, a...

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Outlook's shares crash as wet AMD drug flunks Phase 3 test

After the FDA rejected Outlook Therapeutics’ ophthalmic formulation of bevacizumab last year, the experimental therapy has now failed a late-stage study in patients with a degenerative eye disease. In...

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Advanz Pharma faces revocation of European authorization for liver disease...

The conditional marketing authorization for Advanz Pharma’s liver disease treatment Ocaliva has been revoked following a decision by the General Court of the European Union, the company announced...

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Novartis to cut 139 jobs in NJ as part of commercial refocus

Novartis is laying off 139 employees in East Hanover, NJ, as the company shifts its commercial resources away from two “well-established” medicines. The layoffs will mainly affect members of the...

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Satsuma's NDA filing after CMC woes; Hovione's Irish and US expansion

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FDA is looking into hematology risks after patients take Bluebird's Skysona...

The FDA said Wednesday that it is investigating "serious risk of hematologic malignancy" following the administration of bluebird bio's Skysona, a gene therapy approved for the rare disease known as...

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Applied Therapeutics' rare disease drug gets a CRL

The FDA rejected Applied Therapeutics’ experimental treatment for a rare disease called classic galactosemia, citing deficiencies with the application. The company, while “disappointed with the FDA’s...

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Eli Lilly, Pfizer stress independence of telehealth partners in response to...

In response to lawmakers' concerns, Pfizer and Eli Lilly said last week that they do not influence telehealth partners and their clinicians to prescribe their brand-name drugs. Both companies launched...

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Kiromic and SEC settle over undisclosed clinical trial holds

The SEC said Tuesday that it settled charges filed against Kiromic BioPharma, a tiny immuno-oncology startup based in Texas, for failing to disclose clinical holds on two experimental cancer drugs...

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GSK terminates Phase 2 trial of HBV therapeutic vaccine

GSK has axed a Phase 2 study of a chronic hepatitis B therapeutic vaccine, adding to a string of treatment R&D hurdles for the liver infection. The termination of the study of GSK3528869 was the ...

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Lilly removes body mass ranges from FDA label for weight loss drug

Eli Lilly has removed the commonly used clinical measure of body mass index from the FDA label for its weight loss drug Zepbound, a move that could give physicians greater leeway to decide who should ...

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