FDA

FDA suspends US testing of Sarepta Duchenne drug over safety concerns

The regulator stopped dosing in the U.S. for a drug that’s meant to be a more potent version of Sarepta's marketed medicine Exondys 51, after a patient experienced dangerously low magnesium levels.

By Ben Fidler • June 23, 2022

Bristol Myers CAR-T therapy cleared by FDA for earlier lymphoma use

Breyanzi is now approved in the second-line setting, matching Gilead’s Yescarta, which won a similar clearance from the FDA earlier this year.

By Ned Pagliarulo , Ben Fidler • June 23, 2022

Explore the Trendline➔

The BioPharma Dive Outlook on 2022

After surging into 2021, biotech crashed at the end of last year amid negative clinical trial readouts, ebbing M&A and broader investor pullback. But the year ahead figures to be busy on several fronts and a slate of important studies could deliver good news. 

By BioPharma Dive staff

FDA authorizes Pfizer, Moderna COVID-19 vaccines for young children

The much-anticipated decision follows two days after a panel of independent experts unanimously recommended expanding the shots’ use.

By Ned Pagliarulo • Updated June 19, 2022

FDA advisers recommend Pfizer, Moderna COVID-19 vaccines for youngest children

Independent experts unanimously supported use of both shots in children aged 6 months to 5 years old, clearing the way for an FDA decision.

By Ben Fidler • Updated June 15, 2022

FDA approves Lilly, Incyte drug for alopecia, but includes safety warning

The partners’ JAK inhibitor Olumiant is the first systemic treatment for an autoimmune disease that causes hair loss. But known risks of heart attacks and cancer could complicate its launch. 

By Kristin Jensen • June 14, 2022

Alnylam wins FDA approval of rare disease drug in step toward profitability

Amvuttra, a transthyretin amyloidosis medicine previously known as vutrisiran, is viewed by biotech analysts as important to the company’s goal of ending years of financial losses.

By Ben Fidler • Updated June 14, 2022

FDA staff supportive of Pfizer, Moderna COVID vaccines in young children

Agency advisers will weigh data from the companies at a two-day meeting that begins Tuesday with discussion of Moderna’s shot in children and teens aged 6 to 17 years old. Use in kids under 5 will be debated Wednesday. 

By Jonathan Gardner , Ned Pagliarulo • June 13, 2022

FDA advisers offer unanimous support to second Bluebird gene therapy

By a 13-0 vote, the panel agreed the benefits of Bluebird’s beta thalassemia treatment outweigh its risks, one day after reaching a similar conclusion for another of the company’s drugs.

By Ned Pagliarulo • Updated June 10, 2022

FDA advisers back Novavax's latecomer COVID-19 vaccine

Outside experts debated the shot's safety profile and its efficacy versus the omicron variant but ultimately supported the vaccine, which is made using more traditional technology than Pfizer's and Moderna's.

By Jonathan Gardner • Updated June 7, 2022

FDA extends review of Amylyx ALS drug, delaying approval decision

The agency said it wanted more time to assess additional clinical trial data submitted by Amylyx, and set a new decision date of Sept. 29 for the closely watched treatment. 

By Ned Pagliarulo • June 3, 2022

Pfizer says 3 doses of its COVID-19 vaccine works in youngest children

The drugmaker, along with partner BioNTech, plans to submit the new data to the FDA this week. The agency has scheduled a meeting for outside experts to review the data in mid-June.

By Jonathan Gardner • May 23, 2022

Gilead, having resolved manufacturing issues, forges ahead with HIV drug

The company can now resume testing in nearly a dozen studies that were paused due to FDA concerns about interactions between the drug, lenacapavir, and the glass vials in which it was contained.

By Jacob Bell • May 17, 2022

Lilly wins FDA approval for new kind of diabetes drug

Tirzepatide, now branded as Mounjaro, helped improve blood sugar control in testing and has been shown to have powerful weight loss effects as well, making it one of the most closely watched drugs in Lilly's pipeline.

By Jonathan Gardner • Updated May 16, 2022

BridgeBio sale of FDA voucher good news for Bluebird, other biotech sellers

The drugmaker was able to get $110 million for its voucher, a kind of regulatory fast pass. Other vouchers have sold for similar amounts, boding well for Bluebird, which plans to sell two that it hopes to get this year.

By Ned Pagliarulo • May 13, 2022

With spotlight on FDA, Congress weighs reforms to accelerated drug approvals

The proposed changes could have a notable impact on cell and gene therapy developers, many of which are advancing rare disease therapies that rely on speedy approval pathways.

By Jonathan Gardner • May 10, 2022

Traditional and bespoke registries

In order to truly understand the lived experience of a patient with a given condition or disease, several types of real-world data sources should be analyzed. 

May 9, 2022

FDA places stricter limits on J&J's COVID-19 vaccine after review of rare side effect

The agency is limiting use to adults who either can't or won't take another authorized vaccine after collecting more data on a rare and unusual clotting syndrome that has weighed on the shot's uptake.

By Ben Fidler • May 6, 2022

AstraZeneca and Daiichi set to broaden use of breast cancer drug, challenging Roche

The FDA cleared Enhertu for second-line use in HER2-positive disease, the latest inroads the partners have made against Roche’s dominant portfolio of breast cancer medicines.

By Ben Fidler • May 5, 2022

Spero to lay off 75% of workforce, change strategy after FDA challenges study results

Agency statisticians took a different view of the Phase 3 results the biotech was relying on to support approval of a new antibiotic, an announcement that triggered the sector's latest restructuring.

By Ben Fidler • May 4, 2022

FDA rejects two China-developed cancer drugs

The regulator cited concerns around single-country trials in turning back Hutchmed's pancreatic cancer treatment, while manufacturing issues held up Junshi and Coherus' throat cancer medicine.

By Jonathan Gardner • May 2, 2022

Understanding real-world data in the real world

Real-world data (RWD) and real-world evidence (RWE) are valuable across the entire pharmaceutical product lifecycle, from examining patient outcomes to supporting disease education and awareness and beyond. 

May 2, 2022

Moderna seeks FDA clearance for COVID-19 vaccine in young children

The biotech's shot would become the first available to children under 6, the last remaining age group currently ineligible for vaccination. 

By Kristin Jensen • April 28, 2022

FDA panel supports agency push to raise approval bar for certain cancer drugs

Though agency advisers were hesitant to label an entire drug class, they backed the FDA's interest in demanding randomized trials of PI3 kinase-blocking drugs, a decision that could impact future research.

By Jonathan Gardner • April 22, 2022

GSK to test FDA's tough stance on anemia pills

Unlike treatments from Akebia and FibroGen, GSK's drug hasn't been tied to a higher risk of heart problems, potentially easing the agency's concerns.

By Ben Fidler • April 19, 2022

Troubling data leads a biotech to pull its prized drug from market and FDA review

Early analysis of a key clinical trial showed a potentially increased risk of death in cancer patients who received a combination treatment that included TG Therapeutics' approved medicine, Ukoniq.

By Jacob Bell • April 18, 2022