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

FDA offers former biotech unicorn Intarcia a chance for a panel review

Intarcia, which once held a multibillion-dollar valuation, deflated following the FDA’s 2017 and 2020 rejections of its drug-device combination for diabetes.

By Jonathan Gardner • Feb. 9, 2023

FDA approves Gilead’s Trodelvy for expanded use in breast cancer

The agency’s OK is a win for Gilead, which has bet big on the drug. But the company will face tough competition from AstraZeneca and Daiichi Sankyo’s fast-selling Enhertu.

By Ned Pagliarulo • Feb. 3, 2023

GSK secures FDA approval of anemia drug, but with limitations

The approval is a win for GSK as the FDA had previously rejected two similar drugs from Akebia Therapeutics and Fibrogen. But it comes with restrictions on its use and a safety warning.

By Ned Pagliarulo • Feb. 2, 2023

FDA to convene advisers on review of RSV vaccines from GSK, Pfizer

The agency is currently assessing applications filed by the two companies for what they hope will become the first vaccines against the virus in older adults.

By Delilah Alvarado • Updated Feb. 2, 2023

Head of FDA gene therapy office set to retire in March

Wilson Bryan, a key decision maker in the agency’s oversight of genetic medicines, is expected to leave next month amid an organizational revamp of his office, the FDA confirmed.

By Kristin Jensen • Feb. 1, 2023

FDA approves cancer drugs from Lilly, Menarini

The agency’s clearance of Jaypirca gives Lilly another win from its Loxo buyout, while Menarini Group’s bet on Radius Health’s oral SERD has now paid off with Orserdu’s OK.

By Ned Pagliarulo • Updated Jan. 31, 2023

Merck gets a ‘surprise’ win for Keytruda in early lung cancer

The FDA gave the top-selling immunotherapy a broad label in adjuvant lung cancer despite mixed results in the main supporting study, taking one Wall Street analyst by surprise. 

By Ben Fidler • Jan. 27, 2023

FDA advisers back agency plan to simplify COVID-19 vaccination

The expert committee supported making primary and booster shots the same composition, while recommending periodic updates to better match the vaccines to circulating variants.

By Ned Pagliarulo , Jonathan Gardner • Updated Jan. 27, 2023

FDA advisers back Cidara and Melinta’s antifungal treatment

The drug, if approved, would be the first new treatment in more than a decade for two conditions that stem from Candida fungus.

By Kristin Jensen • Jan. 25, 2023

FDA weighs shift in COVID vaccination strategy

Agency scientists are proposing to update COVID shots once a year to match circulating coronavirus strains, as well as simplifying current vaccination regimens.

By Ned Pagliarulo • Jan. 23, 2023

Why Wall Street shrugged at Lilly’s Alzheimer’s setback

Analysts argue the FDA’s choice to decline accelerated approval for donanemab, while surprising, is not the main factor that will determine whether it eventually becomes a commercial success.

By Jacob Bell • Jan. 20, 2023

FDA lifts hold on Astellas gene therapy for Pompe disease

The regulator’s decision ends a seven-month study pause that followed a report of one participant experiencing mild symptoms of peripheral neuropathy.

By Christopher Newman • Jan. 20, 2023

FDA rejects Lilly Alzheimer’s drug, citing insufficient data

The drugmaker had sought an accelerated approval based on data from a small mid-stage trial. Results from a larger study are due in the second quarter.

By Ned Pagliarulo • Jan. 20, 2023

JPM23: Califf on accelerated approvals, biotech startups’ looming cliff and an explanation from Editas

Venture investors cautioned of a coming funding crunch for young biotechs, while the FDA commissioner shared advice for drugmakers.

By Jacob Bell , Gwendolyn Wu , Ned Pagliarulo • Jan. 10, 2023

5 FDA decisions to watch in the first quarter

An Alzheimer’s drug from Eli Lilly, BioMarin’s hemophilia gene therapy and a new type of breast cancer treatment are among the top decisions on the agency’s docket through the end of March.

By Ben Fidler , Ned Pagliarulo , Jonathan Gardner , Delilah Alvarado • Jan. 9, 2023

FDA approves drugs from Gilead, Roche, TG Therapeutics to close 2022

The agency’s main review office cleared 37 new medicines last year, its lowest total since 2016 and well below 2021’s mark of 50.

By Ned Pagliarulo • Jan. 3, 2023

FDA approval of Biogen Alzheimer’s drug was ‘rife with irregularities,’ congressional report says

An investigation by two House committees concluded the FDA failed to document all its meetings with Biogen, and shifted to consider an accelerated clearance on a “substantially abbreviated timeline.”

By Jonathan Gardner • Dec. 30, 2022

FDA sets decision date for Alvotech’s Humira biosimilar

Along with the review deadline, Alvotech also disclosed the FDA had turned back an application for interchangeability as the agency needs to reinspect a manufacturing facility.

By Jonathan Gardner • Dec. 22, 2022

Europe broadens Lynparza use as FDA ups scrutiny of PARP drugs

Lynparza’s authorization in first-line prostate cancer comes days after U.S. regulators delayed a decision on the same indication, a potential sign of the FDA’s concerns around PARP blockers.

By Ben Fidler • Dec. 21, 2022

Government spending bill would tighten FDA oversight of accelerated drug approvals

The proposed bill would strengthen rules around confirmatory testing, help speed product withdrawals and set up a panel of agency officials to better coordinate policy.

By Jonathan Gardner • Dec. 20, 2022