FDA staff skeptical of Sarepta’s Duchenne gene therapy, documents show

Sarepta has not provided “unambiguous evidence” its treatment can help patients, agency scientists wrote in documents released ahead of a crucial Friday advisory committee meeting.

By Ben Fidler • Updated May 10, 2023

Abortion providers in 3 states sue over mifepristone restrictions

The lawsuit seeks to remove safety restrictions on mifepristone or, alternatively, prevent further altering of access to the drug.

By Sydney Halleman • May 9, 2023

‘The luckiest of the unlucky’: A Duchenne gene therapy brings hope to families — and tests the FDA

The FDA in June approved a treatment that patient advocates and doctors believe to be a breakthrough for a deadly disease, but hasn’t yet proven that it works. 

By Ben Fidler , Shaun Lucas • May 7, 2023

FDA approves first RSV vaccine, clearing GSK’s Arexvy for older adults

The decision represents the first fruits of a scientific breakthrough a decade ago that gave drugmakers, among them GSK, Pfizer and Moderna, a blueprint for an effective shot against the virus.

By Delilah Alvarado • Updated May 3, 2023

FDA clears Pfizer’s pneumococcal vaccine for infants and children

Pfizer’s latest Prevnar shot covers 20 strains of the virus, more than the 13 covered by its previous version and the 15 by Merck’s Vaxneuvance.

By Delilah Alvarado • Updated May 3, 2023

FDA approves Seres’ microbiota drug for recurrent gut infection

The drug is the first microbiota-based pill to win U.S. approval. Like a rival therapy approved in December, it’s for difficult-to-treat C. diff. infections.

By Christopher Newman • Updated April 27, 2023

Supreme Court maintains access to abortion pill, blocking restrictions on its use

The stay suspends a Texas judge’s order that invalidated the FDA’s approval of mifepristone, keeping it available while a circuit court hears the case.

By Delilah Alvarado • Updated April 22, 2023

FDA approves earlier use of Roche’s Polivy in common form of lymphoma

The agency’s decision also converts the biologic drug's approval from accelerated to full, confirming an earlier OK based on tumor responses.

By Jonathan Gardner • April 20, 2023

Supreme Court delays decision in battle over abortion pill

An administrative stay of a Texas district court ruling will remain in place through Friday, pushing out a Supreme Court decision in the closely watched case over the drug mifepristone.

By Delilah Alvarado • April 19, 2023

FDA favors single dose of updated COVID shots in shift to simplify vaccination

The regulator also authorized a second bivalent booster for adults 65 years or older, or those with weak immune systems.

By Delilah Alvarado • April 18, 2023

Manufacturing issues spur FDA to deny approval of Lilly colitis drug

The drugmaker said it is working with the FDA to resolve the issues so it can launch the medicine, an ulcerative colitis treatment known as mirikizumab, “as soon as possible.”

By Christopher Newman • April 14, 2023

Appeals court rules abortion drug can stay on market — but limits access

The Department of Justice appealed the order on Thursday, moving the fight over access to mifepristone to the Supreme Court.

By Delilah Alvarado • Updated April 13, 2023

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 April 8 decision by a district court judge in Texas set off a high-profile chain of litigation that has now reached the Supreme Court, whose decision could carry ripple effects for drug regulation more broadly.

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