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BioMarin's hemophilia gene therapy Roctavian lands FDA nod with 'glimmers' of enthusiasm among doctors

Fierce Pharma

After an initial rejection in 2020 and a review delay earlier this year, BioMarin’s Roctavian has finally got the FDA go-ahead to introduce a gene therapy for a not-so-rare disorder.

Doctors 345
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FDA turns down Outlook's ophthalmic version of Roche's Avastin over manufacturing, data shortfalls

Fierce Pharma

Even without an FDA approval, Roche’s cancer drug Avastin is widely used by doctors to treat certain eye diseases. Even without an FDA approval, Roche’s cancer drug Avastin is widely used by doctors to treat certain eye diseases.

FDA 307
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As FDA puts CAR-T safety under microscope, researchers dig into adverse events record of BCMA therapies

Fierce Pharma

A new study examined side effect reports of BMCA immunotherapies from the FDA Adverse Event Reporting System, hoping to help inform doctors in their treatment decisions. But these immunotherapies also come with various potentially dangerous side effects. |

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US WorldMeds' Iwilfin bags FDA nod as 1st oral maintenance treatment for high-risk neuroblastoma

Fierce Pharma

But now, patients and doctors have a new oral option. | FDA has approved US WorldMeds’ Iwilfin—also known as eflornithine—as a new oral maintenance therapy for high-risk neuroblastoma. While existing neuroblastoma treatments can help patients achieve remission, sustaining that remission has proven tricky.

FDA 265
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Merck's Keytruda hits overall survival goal in early lung cancer as FDA decision nears

Fierce Pharma

What doctors described as a “messy” treatment landscape in early-stage non-small cell lung cancer (NSCLC) could get clearer thanks to a first-in-class overall survival win from Merck & Co.’s s Keytruda.

Doctors 318
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Eisai, Biogen's Leqembi may face rollout hurdles now, but experts still like the Alzheimer's drug

Fierce Pharma

After a full FDA approval last week triggered Medicare coverage of Eisai and Biogen’s Leqembi, doctors are still trying to work out the logistics of testing and reimbursement. But at least two experts remain positive about the drug’s efficacy and safety profile.

Doctors 232
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Pfizer's 'entrenched' tafamidis franchise will be tough to challenge for Alnylam, BridgeBio: analysts

Fierce Pharma

With backing from an FDA advisory committee and results from a positive phase 3 trial, respectively, Alnylam and BridgeBio are each on track to enter the blockbuster transthyretin amyloidosis cardi | Alnylam and BridgeBio are each on track to enter the blockbuster transthyretin amyloidosis cardiomyopathy (ATTR-CM) market.

Doctors 266