5 FDA decisions to watch in the second quarter

2024-04-01

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The year started well for the biotechnology industry. Steady dealmaking coupled with a rebound in initial public offerings helped lift investor confidence and, with it, drugmaker stocks.

While that optimism has moderated somewhat since, a series of decisions by the Food and Drug Administration over the next three months could help the sector sustain its upturn. The agency may expand approvals for two cancer cell therapies and clear a third vaccine for respiratory syncytial virus. Sanofi and Regeneron await a decision on a lucrative new use for their top-selling Dupixent, while Geron could see decades of drug research finally pay off.

Here are five FDA decisions to watch in the second quarter.

CAR-T therapies for early multiple myeloma treatment

The FDA this quarter could broaden the use of two cell therapies for multiple myeloma in decisions potentially worth billions of dollars to their respective developers.

By April 5, the agency will decide whether to clear Johnson & Johnson and Legend Biotech’s Carvykti for use in myeloma patients as early as after one relapse. That decision could be accompanied by a similar OK for Bristol Myers Squibb’s Abecma and open up use of the therapies, which are currently only available in the U.S. for people with more advanced disease.

European regulators have already endorsed earlier use of Abecma and Carvykti based on trial data showing they help people live longer without their disease progressing. But the FDA has appeared less supportive. The agency delayed a decision on Abecma last year and held a March advisory meeting to more closely scrutinize the data for it and Carvykti. In briefing documents, staff scientists flagged an apparent increase in the risk of death early on in the clinical trials of both therapies.

The companies attributed that finding to limitations in the “bridging therapies” study participants could receive while waiting for CAR-T treatment. Panelists were convinced and backed both, agreeing that those issues likely wouldn’t occur as frequently in practice.

Nonetheless, the regulatory scrutiny suggests that the FDA might put new safety restrictions on Carvykti and Abecma if it chooses to clear them for earlier use. — Jonathan Gardner

Moderna’s vaccine for RSV

COVID-19 vaccine makers Pfizer and Moderna have been facing the downside of their pandemic success, as falling sales have brought tough questions from investors.

Moderna, for its part, aims to follow its COVID shot with several other messenger RNA-based vaccines, including one for respiratory syncytial virus that’s now under FDA review.

Respiratory syncytial virus, or RSV, can cause severe illness in infants, older adults and people with compromised immune systems. Two vaccines, from GSK and Pfizer, are already approved to prevent RSV-related disease in adults over the age of 60. Sales of each have been strong, although GSK has established a clear edge.

Moderna argues its vaccine, dubbed mRNA-1345, can compete. The shot is a pre-filled, single-dose vaccine, potentially offering more convenient administration.

In Phase 3 testing, the vaccine was 84% effective at preventing infection with two or more symptoms in older adults. That efficacy does appear to wane over time, however.

At a recent meeting, Centers for Disease Control and Prevention advisers reviewed known safety risks for GSK’s and Pfizer’s shots, both of which were associated in very rare cases with Guillain-Barré syndrome, or GBS, a neurological condition affecting the nerves. Moderna has not reported any cases of GBS in Phase 3 testing of its shot.

An FDA decision on mRNA-1345 is set for May 12. — Delilah Alvarado

Geron’s imetelstat for myelodysplastic syndromes

Drug development is a time-consuming, expensive process. Geron, a biotech founded 34 years ago to pursue stem cell research, knows this well.

The company’s long journey may finally reach an end by June 16, when the FDA will decide whether to approve a bone marrow disease drug Geron has been developing for nearly two decades. Called imetelstat, the drug treats myelodysplastic syndromes and some analysts believe it has blockbuster potential.

Over the many years of Geron’s research, imetelstat failed studies in multiple cancer types, and the company lost Johnson & Johnson as a partner along the way. But the drug finally succeeded in Phase 3 testing for myelodysplastic syndromes last year. And in March, it secured the endorsement of an FDA advisory panel despite skepticism from agency reviewers.

Geron aims to challenge Bristol Myers Squibb’s Reblozyl, first with a clearance in people with “lower-risk” disease and then in more people who have the condition. Stifel analyst Stephen Wiley estimated the drug could generate $2 billion in peak yearly sales, as it could be helpful in people who don’t respond well to Reblozyl.

Geron could be a buyout target, too, according to Wiley, though the company has said it plans to sell the drug on its own. — Ben Fidler

Sanofi and Regeneron’s Dupixent for COPD

Dupixent is one of the pharmaceutical industry’s most widely used and lucrative products. Approved for asthma, eczema and three other inflammatory conditions, the drug earned the companies more than $11 billion in sales last year.

By June 27, the FDA could add another major indication to Dupixent’s list of approved uses. The regulator is currently reviewing the injectable medicine as a treatment for a type of chronic obstructive pulmonary disease, or COPD.

If cleared by the FDA, Dupixent would be the first biologic drug for people with moderate-to-severe COPD who also have evidence of what’s called Type 2 inflammation. A pair of late-stage studies, which enrolled nearly 1,900 people, showed Dupixent could cut the rate of COPD attacks by about one-third. Analysts therefore expect the FDA to issue an approval and forecast billions of dollars in additional yearly sales. — Ned Pagliarulo

Pfizer’s gene therapy for hemophilia B

Some time during the second quarter, the FDA will decide whether to clear a gene therapy from Pfizer for hemophilia B, the less common form of the blood clotting disorder.

Reaching this point has been a long time coming for Pfizer, which licensed the therapy from Spark Therapeutics a decade ago. In subsequent testing, treatment lowered bleeding rates and reduced the need for regular blood transfusions — powerful benefits meant to last many years with a single infusion.

But it’s unclear whether Pfizer will find a market if the FDA chooses to approve its therapy. A rival genetic medicine, from CSL Behring and UniQure, won U.S. approval for hemophilia B in November 2022. However, few patients have been treated to date, sluggish uptake that could reflect the breadth of effective options available.

The story is similar with Roctavian, a gene therapy BioMarin Pharmaceutical sells for the more common “A” form of hemophilia. Only three patients received Roctavian last year: two in Germany and one in the U.S.

Pfizer’s therapy, which in January won Canadian approval, could provide another data point. But the company seems to be downplaying its potential. In recent presentations for investors, a once-weekly shot called marstacimab has gotten higher billing. — Ned Pagliarulo