Mobocertinib

The FDA's accelerated approval pathway, enacted in 1992, has led to over 200 drugs reaching market based on surrogate endpoints. Despite recent withdrawals like Pfizer's Oxbryta and Takeda's Exkivity, the program has a strong track record, with over half of approvals converting to full approval. Challenges include failed confirmatory trials and the need for better biomarker understanding.

Accelerated approval, despite recent controversies, has historically provided therapeutic momentum, particularly in HIV and cancer treatments. Key issues include reliance on surrogate endpoints and confirmatory trial failures, yet the pathway has led to significant drug approvals, offering hope for diseases with high unmet needs.

Pfizer's sickle cell disease therapy Oxbryta was pulled from market due to risks, highlighting the challenges of accelerated approval. The FDA's program has brought nearly 300 drugs to market, often years earlier, but success rates vary. While many drugs have secured traditional approval, others like Oxbryta and Aduhelm were ultimately unsuccessful. The pathway relies on biomarkers predicting clinical benefit, but confirmatory trials are often delayed, raising concerns about safety and efficacy. Experts argue for tighter deadlines and better validation of biomarkers to improve the program's reliability.

Pfizer withdrew Oxbryta from global markets due to increased risk of death, joining other drugs like Aduhelm, Exkivity, Ukoniq, and Zydelig that were approved under the FDA's accelerated pathway but later withdrawn. The pathway, implemented in 1992, has helped bring nearly 300 drugs to market but faces scrutiny over delayed confirmatory trials and surrogate endpoints. Sarepta's Elevidys is an exception, securing full approval despite missing primary efficacy endpoints.

The global cancer biomarkers market size was USD 31.90 billion in 2023 and is projected to reach USD 107.33 billion by 2033, growing at a CAGR of 12.9%. Key drivers include rising technical developments, individualized treatment approaches, and increasing prevalence of cancer globally. The breast cancer segment led the market in 2023, while the genetic biomarkers segment dominated in terms of revenue. North America held the largest market share in 2023, with the U.S. leading due to high prevalence of renal cancer and strong pipeline. Technological innovations and advancements in personalized medicine are expected to further fuel market growth.