FDA pitches faster path to rare-disease therapies
The FDA, led by Dr. Marty Makary in a White House event with President Trump, proposed a new framework to speed approvals for therapies targeting rare diseases.
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Drug Approvals
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One-pivotal-trial rule aims to redefine drug approvals
FDA officials say a single pivotal trial will be the default standard for drug approvals, a policy shift outlined in an NEJM perspective by Marty Makary and Vinay Prasad.
One-Study Path to FDA Drug Approvals Sparks Policy Debate
FDA Commissioner Marty Makary and Prasad argue that a single pivotal study could suffice for drug approvals, a shift aimed at speeding access but drawing concerns about the robustness of evidence and patient safety.
FDA’s Fast-Track Drug Vouchers Spark Sign-Off Scrutiny
AP reports that FDA Commissioner Marty Makary’s voucher-based effort to accelerate drug approvals into a one-month window has stirred alarm inside the agency. Questions swirl over who has legal authority to sign off on these expedited approvals, with senior officials and political appointees increasingly involved in the process, diverging from the traditional scientist-led review. The program, not codified in federal rules and now expanded to 18 vouchers, has coincided with staff layoffs and leadership turnover, fueling concerns about politicization, safety standards, and potential legal risk for the agency as critics warn that such rapid reviews lack scientific precedent.
FDA Disarray Sparks Staff Concerns and Credibility Issues
The FDA is experiencing significant turmoil with high-profile staff departures, leadership resignations, and internal mistrust, raising concerns about its reliability and impact on drug approvals and public health, amid broader government downsizing and political pressures.
"2023's Remarkable Medical Breakthroughs: Drug Approvals, Vaccines, and Innovations"
GoodRx Health has compiled a list of the most notable drug and vaccine approvals of 2023, selected by pharmacists. The list includes Paxlovid for COVID-19, Arexvy, Abrysvo, and Beyfortus for RSV, Opill for contraception, Zurzuvae for postpartum depression, Leqembi for Alzheimer's disease, OTC naloxone for opioid overdoses, Zepbound for chronic weight management, Roctavian for hemophilia A, and Casgevy and Lyfgenia for sickle cell disease. These approvals are seen as meaningful steps forward in improving human health and accessibility to treatments.
"Pfizer's Hidden Potential: Uncovering the Undervalued Gem on NYSE"
Pfizer's financial position is expected to benefit from recent events, including multiple drug and vaccine approvals, which will help offset potential revenue losses from exclusivity and reduced demand for the COVID-19 vaccine. The company's experimental drug, danuglipron, is poised to compete with leaders in the diabetes treatment market. Additionally, the FDA approval of Abrysvo for respiratory syncytial virus in older people opens up significant commercial opportunities. The recent FDA approvals of Litfulo and Ngenla further strengthen Pfizer's position in the pharmaceutical industry. Ngenla's entry into the US market is expected to drive strong sales growth and profit generation. With extensive long-term data and market recognition, Pfizer aims to regain its share of the global human growth hormone market. Despite a recent decrease in share price, technical analysis suggests a potential reversal upwards. The article concludes with an "outperform" rating for Pfizer.
Controversy Surrounds Abortion Pill Decision and FDA Support
Pfizer CEO Albert Bourla signed a letter along with over 200 pharmaceutical company executives in support of the FDA's authority to regulate drugs after a federal judge in Texas suspended the agency's approval of the abortion pill mifepristone. The executives raised concerns that the ruling will set a precedent for diminishing the FDA's authority over drug approvals, which would create uncertainty for the entire industry. The dueling orders by two federal judges create a complicated legal standoff that could potentially escalate to the Supreme Court.