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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 320
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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 275
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House lawmakers launch probe into FDA's response to nationwide drug shortages

Fierce Pharma

As shortages of drugs like amoxicillin, penicillin, Adderall and certain chemotherapies continue to confound doctors and patients in the U.S., two high-ranking Republican lawmakers are pressing the | As shortages of drugs like amoxicillin, penicillin, Adderall and certain chemotherapies continue to confound doctors and patients in the U.S.,

FDA 230
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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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Roche scores FDA nod for prefilled syringe version of Vabysmo, easing its administration

Fierce Pharma

Roche has taken another step in its advancement of the blockbuster medicine, scoring an FDA nod to provide it in a prefilled syringe. The 6-mg, single-dose shot will give doctors a simplified, ready-to-use alternative to extracting Vabysmo from a vial.

FDA 227
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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 283
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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 232