FDA, citing safety concerns, places partial hold on Merck KGaA’s MS drug

Evidence of possible liver damage among study participants adds to growing concerns about the potential of BTK inhibitors to treat MS, a strategy being pursued by several large drugmakers. 

By Kristin Jensen • April 12, 2023

Biotech fears ‘dangerous’ precedent as judge challenges FDA authority

Industry leaders warned that “any medicine is at risk” if a federal judge’s decision to overturn the approval of abortion drug mifepristone is upheld.

By Ben Fidler , Delilah Alvarado • April 10, 2023

Federal judge invalidates FDA approval of abortion pill

The decision imperils access to the drug more than two decades after its U.S. approval, and could carry consequences for the FDA’s authority over prescription medicines.

By Delilah Alvarado • April 8, 2023

FDA withdraws preterm birth drug’s approval, denying maker’s request for delay

Commissioner Robert Califf and Chief Scientist Namandjé Bumpus determined there was “no justification” for keeping Covis Pharma’s controversial treatment Makena on the market.

By Ned Pagliarulo • April 6, 2023

Vertex, CRISPR finish US filing for gene editing drug approval

The approval application is the first in the U.S. for a CRISPR-based medicine and puts the partners ahead of a rival therapy from Bluebird bio. 

By Christopher Newman • April 3, 2023

5 FDA decisions to watch in the second quarter

By the end of June, the agency could clear a gene therapy for Duchenne muscular dystrophy and two vaccines for RSV, as well as issue a precedent-setting decision on a closely watched ALS drug.

By Ben Fidler , Christopher Newman , Delilah Alvarado , Jacob Bell • April 3, 2023

FDA approves Narcan for over-the-counter use

It’s the first time a naloxone-based therapy for opioid overdoses has been cleared for use without a prescription, potentially helping to improve access.

By Delilah Alvarado • March 30, 2023

BrainStorm files ALS drug application over FDA protest

The medicine, a personalized stem cell treatment, will be reviewed by an FDA advisory committee sometime in the future, the company said.

By Ned Pagliarulo • March 27, 2023

FDA approves new antifungal as concerns grow over drug-resistant fungus

Rezzayo is the first new treatment for candidemia and invasive candidiasis to win FDA approval in years.

By Kristin Jensen • March 23, 2023

FDA advisers support conditional clearance of Biogen’s ALS drug

Though the drug, called tofersen, missed the main goal of a key clinical trial, it showed an effect on a protein tied to ALS. Experts see that as a good sign, but some worry Biogen has not yet gathered enough supportive evidence.

By Jacob Bell • March 22, 2023

FDA staff signal support for Biogen ALS drug, but highlight data hurdles

In documents released Monday, agency staff appeared inclined to approve Biogen’s tofersen, which failed in its main trial. Gathering confirmatory evidence of its benefit could be difficult, however.

By Jacob Bell , Ned Pagliarulo • Updated March 20, 2023

FDA reversal sets up high-stakes hearing on Sarepta gene therapy

The regulator will convene a panel of advisers to consult on an approval decision for the biotech’s Duchenne treatment, a change from just a few weeks ago when Sarepta had said a meeting wouldn’t be held.

By Ned Pagliarulo • March 17, 2023

FDA advisers back earlier use of Roche lymphoma drug

The positive vote comes despite doubts from FDA scientists and increases the chance that Polivy could soon be approved as a first-line treatment alongside a drug regimen called R-CHOP.    

By Jonathan Gardner • March 10, 2023

Pfizer’s Biohaven deal pays dividends with new drug approval

The medicine, a nasal spray for migraines to be sold as Zavzpret, was a key part of Pfizer’s roughly $12 billion buyout of Biohaven last year. 

By Ben Fidler • March 10, 2023

Controversial drug for preterm birth to be withdrawn, maker says

The decision by Covis Pharma closes a yearslong battle over continued use of Makena, the only treatment available for prevention of early labor.

By Delilah Alvarado • Updated March 8, 2023

BioMarin, as expected, sees delay to FDA review of hemophilia gene therapy

The agency is considering three-year follow-up data in its review of Roctavian, pushing back the potential U.S. arrival of BioMarin’s drug once again.

By Kristin Jensen • March 7, 2023

FDA decision on preterm birth drug’s withdrawal nears, putting spotlight on patients, agency

A yearslong regulatory battle over the hormonal shot Makena is approaching its end, with consequences for both preterm birth prevention and the agency’s authority to withdraw drugs shown to be ineffective in follow-up testing.

By Delilah Alvarado • March 6, 2023

GSK’s RSV vaccine wins FDA panel backing, matching Pfizer

Committee members appeared more confident in their recommendation of GSK's shot, with fewer dissents in separate votes on its safety and effectiveness.

By Delilah Alvarado • March 2, 2023

FDA asks advisers to review Lynparza amid growing scrutiny of PARP drugs

The meeting to discuss Lynparza's use as a frontline treatment for prostate cancer follows a string of withdrawals for drugs in its class, and evidence it didn’t meaningfully extend survival in late-stage testing.

By Jonathan Gardner • March 2, 2023

Pfizer’s RSV vaccine gets lukewarm backing of FDA advisers

The panel voted 7-4 in favor of Pfizer’s shot, but also expressed concerns over its safety profile and what they viewed as holes in the company’s data.

By Delilah Alvarado • March 1, 2023

FDA approves Reata rare disease drug in controversial decision

The clearance, the first for a medicine for Friedreich’s ataxia, comes after years of questions about its benefit and sent the drugmaker’s shares soaring.

By Kristin Jensen • March 1, 2023

FDA rejects Cytokinetics’ heart failure drug

The biotech said the FDA was unconvinced by available study evidence and asked for data from an additional clinical trial, which the company currently does not plan to run.

By Christopher Newman • Feb. 28, 2023

Billy Dunn, FDA official in charge of neuroscience drug reviews, to leave agency

Dunn had become a controversial figure after his role in the FDA's review and approval of Biogen's Alzheimer's drug Aduhelm.

By Ned Pagliarulo • Updated Feb. 27, 2023

CMS plans trio of experiments aimed at lowering drug costs

The pilot programs could allow adjusted payments for drugs cleared under accelerated approval and help states manage the costs of gene therapies.

By Christopher Newman • Feb. 15, 2023

GSK wins full approval for cancer immunotherapy, looks to expand use

The drugmaker converted Jemperli’s accelerated approval in endometrial cancer, while outside advisers endorsed its plan to study the Keytruda rival in rectal tumors.

By Jonathan Gardner • Feb. 10, 2023