Five Key FDA Approval Decisions to Watch in Q2 2024

In 2024, the biotechnology industry got off to a good start. Stable trading combined with a rebound in initial public offerings (IPOs) indicates that the industry is recovering, helping to boost investor confidence.

Although this optimism has since weakened somewhat, a series of decisions by the U.S. FDA in the next three months could help the industry continue to improve. The FDA may approve applications for expanded indications for two cell therapies and might also approve the world's third RSV vaccine. Sanofi and Regeneron are awaiting a decision on the approval of a new indication for their best-selling autoimmune monoclonal antibody drug Dupixent, while Geron is poised to see decades of drug research and development finally pay off.

Industry media BioPharma Dive summarized five FDA approval decisions worth watching in the second quarter of 2024., these decisions are also sufficient to reflect the latest trends in the industry.

FDA May Expand Indications for Two CAR-T Cell Therapies to Treat Multiple Myeloma in Q2; If Approved, This Decision Could Bring Billions of Dollars in Commercial Value to Relevant Pharmaceutical Companies.

By April 5,FDA to Decide on Approval of Johnson & Johnson and Legend Biotech's CAR-T Therapy Carvykti for Early Treatment of Relapsed Multiple MyelomaAt the same time this decision was made,BMS's Abecma is also expected to receive the same approval.This will further expand the use of these therapies, which previously were only available in the U.S. for patients with advanced multiple myeloma.

Currently, as clinical trial data shows that CAR-T therapy can help patients extend their lives without further disease progression, the European regulatory agency EMA has approved the use of Abecma and Carvykti in earlier-stage patients. However, the FDA appears less supportive of this decision. Last year, the FDA delayed its decision on expanding the use of Abecma for earlier-stage multiple myeloma patients and held a consultative meeting in March to more carefully review the clinical data of Abecma and Carvykti. In briefing documents, scientists noted a significantly increased risk of early mortality in the clinical trials of both therapies.

The two companies attributed this phenomenon to the limitations of "bridging therapies" that clinical patients may receive while waiting for CAR-T treatment. In its recent review, the FDA expressed confidence in and support for this view, unanimously agreeing that these issues are unlikely to occur frequently in real-world clinical settings.

Nevertheless, regulatory reviews suggest that if the FDA chooses to approve the early use of Carvykti and Abecma, it might impose new safety restrictions and regulations on them.

Pfizer and Moderna have been facing the negative repercussions following the successful development of the COVID-19 vaccine, with the decline in Comirnaty sales sparking sharp questions from investors.

In the case of Moderna, its goal is to roll out several other mRNA-based vaccines after the COVID-19 vaccine, including a respiratory syncytial virus vaccine currently under FDA review.

Respiratory Syncytial Virus (RSV) can cause severe illness in infants, the elderly, and individuals with compromised immune systems. Currently, two RSV vaccines developed by GSK and Pfizer have been approved for the prevention of RSV-related diseases in adults aged 60 and above. Despite GSK's clear advantage, sales of both RSV vaccines remain robust.

Moderna believes that its vaccine, named mRNA-1345, is also competitive. This vaccine is a pre-filled single-dose vaccine that may provide more convenient disease management.In Phase III clinical trials, the vaccine was 84% effective in preventing elderly individuals from developing two or more infection symptoms. However, over time, this efficacy does appear to diminish.

At a recent meeting, advisors to the U.S. Centers for Disease Control and Prevention reviewed the known safety risks of GSK and Pfizer's RSV vaccines, both of which have been linked to Guillain-Barré Syndrome (GBS), a neurological disorder, in extremely rare cases. Currently, Moderna has not reported any GBS cases in the Phase III clinical trial of its vaccine.FDA to Make Regulatory Decision on mRNA-1345 on May 12

Drug development is a time-consuming and costly process. Geron, a biotechnology company founded 34 years ago, is dedicated to stem cell research.

The company's long journey of new drug development may finally come to an end on June 16, when the FDA will decide whether to approve Geron's new drug for bone marrow diseases, which has been under development for nearly two decades. The drug, named imetelstat, is used to treat myelodysplastic syndromes, and some analysts believe it has blockbuster potential.

Over the years of clinical research at Geron, imetelstat has failed in studies across various cancer types, and along the way, the company lost its partnership with Johnson & Johnson. However, last year, the drug finally succeeded in a Phase III trial for myelodysplastic syndromes. In March this year, despite skepticism from FDA reviewers, it still received endorsement from the FDA advisory panel.

Geron Aims to Challenge BMS's ReblozylReblozyl Outperforms Epogen, a Standard Drug with Decades of History, in Treating Anemia Caused by Myelodysplastic Syndromes. In this regard, Stifel analyst Stephen Wiley estimates that the annual sales peak of imetelstat could reach $2 billion, as it may benefit those who respond poorly to Reblozyl. Wiley stated that Geron could also become an acquisition target, although the company has expressed its plan to market the drug independently.

Dupixent is now one of the most widely used and highest-selling products in the pharmaceutical industry. The drug is approved for the treatment of asthma, eczema, and three other inflammatory diseases, generating over $11 billion in sales last year for Sanofi and Regeneron.

By June 27, the FDA is likely to add another major indication to the approved indications list for Dupixent — chronic obstructive pulmonary disease (COPD), as the regulatory agency is currently reviewing the marketing application for Dupixent in treating COPD.

If approved by the FDA, Dupixent will become the first biologic drug for patients with moderate to severe chronic obstructive pulmonary disease.These patients also have evidence of so-called Type 2 inflammation. Two late-stage clinical trials involving nearly 1,900 participants demonstrated that Dupixent could reduce the incidence of COPD by approximately one-third. As a result, analysts anticipate that the FDA will approve the product and predict that its annual sales will increase by billions of dollars.

In the second quarter, the FDA will decide whether to approve Pfizer's gene therapy for hemophilia B, a less common blood clotting disorder.

For Pfizer, achieving this goal has been a long time coming. Pfizer acquired the license for this therapy from Spark Therapeutics a decade ago. Subsequent clinical studies have shown that the drug reduces bleeding rates and decreases the need for regular blood transfusions. A single infusion can bring significant benefits to patients, lasting for many years.

But it is not yet clear,Whether Pfizer will find a market if the FDA chooses to approve its therapy.Because the gene therapy jointly developed by CSL Behring and UniQure has already received FDA approval in November 2022 for the treatment of Hemophilia B. However, so far, very few patients have received the treatment, and the slow uptake of the drug may reflect issues with its accessibility.

This story is similar to Roctavian, a gene therapy sold by BioMarin Pharmaceutical for the treatment of the more common Hemophilia A. However, last year, only three patients received Roctavian treatment—two in Germany and one in the United States.

Currently, Pfizer's therapy has already received approval from Canada in January, which can provide more data support. However, the company seems to be downplaying the drug’s therapeutic potential. This is because, during a recent investor meeting, Pfizer’s marstacimab, a hemophilia B drug that only requires once-weekly injections, achieved higher sales after being approved.