FDA approves Krystal gene therapy for rare wound disorder

Cleared to treat dystrophic epidermolysis bullosa, Vyjuvek is the sixth gene therapy for an inherited disease to gain clearance in the U.S.

By Delilah Alvarado • May 22, 2023

Future of Intercept’s NASH drug in doubt after FDA panel rejection

The safety risks of obeticholic acid were too great for an advisory committee to recommend approval without clear proof of its benefits. But collecting that data may not be “economically feasible,” an executive said.

By Ben Fidler • Updated May 22, 2023

FDA advisers back maternal use of Pfizer RSV vaccine

The panel voted 14-0 that Pfizer’s data showed its shot to be effective. The vaccine could be the first for protecting infants by maternal immunization.

By Christopher Newman • May 19, 2023

FDA staff unsure NASH drug’s ‘modest’ benefits outweigh safety risks

Staff scientists are concerned Intercept Pharmaceuticals’ drug — which could be the first for non-alcoholic steatohepatitis — may cause liver damage and other health problems, documents show.

By Ben Fidler • May 17, 2023

FDA panel narrowly backs approval of Sarepta’s Duchenne gene therapy

Shares in Sarepta rose by 25% Monday following a close advisory committee vote supporting accelerated approval of the biotech’s treatment, despite uncertainty around its benefit.

By Ned Pagliarulo , Ben Fidler • Updated May 12, 2023

FDA advisers back over-the-counter use of birth control pill

The committee members urged the agency to move quickly to improve access to Perrigo’s contraception pill.

By Kristin Jensen • May 11, 2023

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