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Accelerated Approval

All articles tagged with #accelerated approval

health-medicine1 year ago

"Study Reveals Lack of Evidence for Many Accelerated Approval Cancer Drugs"

A study found that most cancer drugs granted accelerated approval by the FDA do not demonstrate clinical benefits within five years, despite the program being intended to give patients early access to promising drugs. Between 2013 and 2017, 63% of cancer drugs granted accelerated approval were converted to regular approval, even though only 43% demonstrated a clinical benefit in confirmatory trials. The study raises concerns about whether patients understand the uncertainty surrounding drugs with accelerated approval and emphasizes the importance of doctors carefully explaining the evidence to patients. Congress recently updated the program, giving the FDA more authority and streamlining the process for withdrawing drugs when companies don’t meet their commitments.

via The Associated Press|
#accelerated-approval#cancer-drugs#clinical-trials
healthcare1 year ago

"Moderna's Push for Accelerated Approval of Cancer Vaccine Hinges on Three Key Factors"

Moderna is aiming for an accelerated approval for its cancer vaccine in partnership with Merck by 2025, following promising Phase II data showing the potential of their individualized neoantigen therapy in reducing the risk of cancer recurrence or death in melanoma patients when used with Merck’s Keytruda. However, the company is waiting on three key developments before seeking accelerated approval. The therapy is currently undergoing Phase III trials in adjuvant melanoma and non-small cell lung cancer.

via Endpoints News|
#accelerated-approval#cancer-vaccine#healthcare
health2 years ago

Desperate Mother's Plea: FDA, Let Me Save My Dying Son

Families of children with Sanfilippo syndrome, a rare genetic condition that causes severe physical and cognitive decline, are urging the U.S. Food and Drug Administration (FDA) to grant accelerated approval to potential treatments. Sanfilippo syndrome has no cure, and many children do not survive past their teenage years. While some treatments have shown promise, the lack of accelerated approval hinders access to these treatments. Families and biotech companies argue that without accelerated approval, funding for research will decline, and progress will be lost. The FDA's Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on surrogate endpoints.

via The Washington Post|
#accelerated-approval#fda#health
biotechhealthcare2 years ago

Rocket Pharmaceuticals Secures FDA Approval and Funding for Gene Therapy Study

Rocket Pharmaceuticals plans to raise $175 million through a stock sale after the FDA agreed to its proposal for accelerated approval of its gene therapy for Danon disease based on a 12-patient study. The company's stock surged by 42% following the announcement. Danon disease is a rare X-linked disorder that weakens the heart and skeletal muscles, with males typically living until around 20 years old and females until around 35 or 40. Rocket's treatment delivers a functional version of the LAMP2 gene using a viral capsid called an AAV.

via Endpoints News|
#accelerated-approval#biotechhealthcare#danon-disease
healthcare2 years ago

FDA Delays Decision on Sarepta's Highly Anticipated Gene Therapy, Shares Tumble.

The FDA has delayed its decision on the approval of Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy by one month, with a new decision expected by June 22. The FDA is also considering granting accelerated approval for the therapy, but only for 4- and 5-year-old patients, rather than all Duchenne patients who could still walk as requested by Sarepta.

via STAT|
#accelerated-approval#duchenne-muscular-dystrophy#fda
health2 years ago

FDA approves drug for rare form of ALS.

The FDA has approved Biogen's injectable drug, tofersen, for a rare genetic mutation that causes ALS, or Lou Gehrig's disease, which affects less than 500 people in the US. The drug is the first for an inherited form of ALS and was approved via the FDA's accelerated pathway, which allows drugs to launch based on promising early results. However, the FDA is requiring Biogen to continue studying the drug in a trial of people who carry the genetic mutation but do not yet have ALS symptoms.

via The Associated Press|
#accelerated-approval#biogen#fda
healthcare2 years ago

FDA Aims to Improve Accelerated Approval Process for Gene Therapies and Oncology Clinical Trials

The FDA has issued draft guidance aimed at improving oncology clinical trials for accelerated approval applications. The guidance discusses the design of clinical trials and ways to improve the data available at the time of accelerated approval and reduce clinical uncertainty for patients by initiating postmarketing confirmatory studies in a timely manner. The draft guidance also provides considerations for sponsors to determine the adequacy of single-arm studies to support an application.

via FDA.gov|
#accelerated-approval#cancer#clinical-trials
healthcare2 years ago

FDA Pushes for Accelerated Approval of Gene Therapies

The head of the Center for Biologics Evaluation and Research at the FDA, Peter Marks, has said that the agency needs to start using accelerated approval to advance gene therapies for rare diseases. Marks said that the FDA can't be so careful about approvals under accelerated approval that it prevents potentially lifesaving therapies from getting to market in a timely manner.

via STAT|
#accelerated-approval#fda#gene-therapy
business2 years ago

Sarepta's Gene Therapy Faces FDA Setback, Stock Plummets.

Sarepta Therapeutics' stock crashed nearly 20% after the FDA announced it would hold an advisory committee to discuss the risks and benefits of its gene therapy for Duchenne muscular dystrophy, despite the company's belief that it had met the requirements for accelerated approval. The FDA's decision to hold the advisory committee meeting before the May 29 approval decision date has raised concerns about a potential extension of the approval date. However, some analysts remain optimistic about the potential for Sarepta's gene therapy to ultimately gain approval.

via Investor's Business Daily|
#accelerated-approval#advisory-committee#business