Tasimelteon

On Friday, the EMA's drug advisory panel issued its latest raft of recommendations and regulatory updates, unveiling positive opinions for five new medicines.Heading the list was a recommendation to approve Amgen's Imdylltra (tarlatamab) to treat adults with extensive-stage small-cell lung cancer (ES-SCLC) whose disease relapsed during or after an initial treatment with platinum-based chemotherapy.The T-cell engager, which targets both DLL3 and CD3, was authorised in the same indication in the US in 2024, where it is marketed as Imdelltra. While the FDA's accelerated approval was based on the Phase II DeLLphi-301 study, the EMA's decision was supported by findings from the Phase III DeLLphi-304 trial. Data reviewed by the agency's Committee for Medicinal Products for Human Use (CHMP) showed that Imdylltra was associated with a significant improvement in median overall survival of 13.6 months, versus 8.3 months for standard care, corresponding to a 40% reduction in the risk of death. Meanwhile, median progression-free survival in the two groups was 4.2 months and 3.2 months, respectively.Similar to the US — where Amgen's drug carries a boxed warning for cytokine release syndrome (CRS) and neurologic toxicities — the EMA said that the product information for Amgen's drug will describe these risks and guidance on their management. Staying in ES-SCLC, the CHMP also recommended approval of PharmaMar's Zepzelca (lurbinectedin) for the maintenance treatment of patients whose disease has not progressed after first-line induction therapy. The alkylating agent, which was recently cleared for this use in the US, is indicated in combination with Roche's anti–PD-L1 Tecentriq (atezolizumab).Thumbs up for Sanofi's SC SarclisaAlso earning itself a CHMP nod was Sanofi and its on-body injector subcutaneous (SC) formulation of Sarclisa (isatuximab). The recommendation covers all four of the multiple myeloma indications currently held by the intravenous (IV) version of the anti-CD38 mAb in the EU.The automated hands-free device uses a 30-gauge hidden and retractable needle that is thinner than commonly used SC injection needles.At the American Society of Clinical Oncology (ASCO) meeting last year, Sanofi shared data from the Phase III IRAKLIA study showing that injectable Sarclisa achieved a 71.1% objective response rate after a median follow-up of 12 months, compared to 70.5% for the IV formulation, meeting the non-inferiority primary endpoint. Progression-free survival rates at 12 months were similar between the two formulations at 66.1% and 65.1%, respectively.Decisions go the way of Ferring, PharmingIn other decisions, the CHMP recommended conditional approval of Ferring Pharmaceuticals' gene therapy Adstiladrin (nadofaragene firadenovec) to treat adults with Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumours.Adstiladrin has been on the market in the US for several years after winning FDA approval at the end of 2022.The CHMP also backed authorisation of Pharming's Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older weighing 45 kg or more. While the oral, selective PI3Kδ inhibitor is approved for this patient group in the US, efforts to expand use into children aged four to 11 years with the rare and progressive primary immunodeficiency were recently rejected by the FDA. Although Pharming is hopeful of addressing the US regulator's issues — which relate to clinical pharmacology and batch testing methodology — the paediatric approval had been expected to drive a Joenja growth inflection this year.Elsewhere, the CHMP issued a positive opinion recommending paediatric use of Proveca Pharma's Bopediat (furosemide) to treat oedema of cardiac or renal origin, oedema of hepatic origin and hypertension in children from birth to under 18 years of age with chronic kidney disease. Bopediat is a hybrid medicine of Lasilix Faible (furosemide), which has been authorised in France since 1977. The EMA noted that Proveca's drug contains the same active substance as Lasilix Faible, but it is available as orodispersible tablets and at a lower strength so that it can be given to young children.Re-examinations end badlyThe EMA on Friday also confirmed the recent decision by Anavex Life Sciences to withdraw an application for its Alzheimer's drug candidate blarcamesine. Having recommended refusing approval at the end of last year, the CHMP was in the process of re-examining the application — as requested by the drugmaker — when the company opted to pull the filing.Another re-examination resulting in a negative outcome went to Hetlioz (tasimelteon) and its developer Vanda Pharmaceuticals, who was seeking to expand use into people with a rare hereditary disorder, Smith-Magenis syndrome. The melatonin‑receptor agonist is already approved in the EU to treat totally blind adults with non-24-hour sleep-wake disorder.Having initially rejected use of Hetlioz in Smith-Magenis syndrome last November, the CHMP reaffirmed its decision at an extraordinary meeting on March 16. The committee expressed concerns about the study used to support the application, including the statistical analysis of the results and the way the trial had been conducted, which led to uncertainties about the drug's effects.Tecovirimat use restrictedThe EMA also said Friday that SIGA Technologies' antiviral tecovirimat should no longer be used for mpox. The drug, also known as TPOXX, was approved in the EU in 2022 to treat smallpox, mpox and cowpox based on data from animal studies.Subsequent trials conducted in humans — including the STOMP study sponsored by the US National Institutes of Health (NIH) — showed that tecovirimat did not heal lesions faster compared with placebo in people presenting with active mpox lesions, while it also failed to improve other outcomes, such as relieving pain or helping to clear the virus from the body faster.

WASHINGTON, March 26, 2026 /PRNewswire/ -- Today, the Taxpayers Protection Alliance (TPA) released a report, "Blocking Breakthroughs: Delays and Denials at the FDA," highlighting the many issues with the Food and Drug Administration's (FDA's) drug approval process. The report comes amid continued controversy over the agency insisting on unrealistic and ethically-fraught criteria for approvals and walking back a bizarre decision to refuse to examine an application by Moderna for its first mRNA seasonal flu vaccine. Fortunately, FDA Center for Biologics Evaluation and Research Director Vinay Prasad—who played a key role in these decisions—will soon be leaving the agency. Key report findings include: FDA drug approval costs have increased significantly over the past ten years, skyrocketing from roughly $30 million per medication in 2015 to more than $50 million in 2025. This only includes administrative costs borne by the agency—and taxpayers and drug sponsors by extension—and does not include billions of dollars in clinical trial expenses. Even as the FDA has more taxpayer dollars and user fees at its disposal, rejections are becoming increasingly common. The 2025 rejection rate of nearly 30 percent was near decade highs. The FDA's approach to five medications in particular (Ebvallo, ONS-5010, High-Dose Spinraza, Hetlioz, and Gefapixant) reflects continued risk aversion that harms consumers. Ross Marchand, Executive Director of the Taxpayers Protection Alliance, offered the following comment: "For too long, the FDA has stood in between patients and life-saving treatments. New leaders at the helm of the Department of Health and Human Services (HHS) and FDA, such as Robert F. Kennedy Jr. and Dr. Marty Makary, have promised to make America healthier and bolster patient access to care. This report shows that is simply not happening, and millions of Americans are paying the price. Bureaucrats need to get out of the way and put patients first. "There are plenty of steps the FDA and Congress can take to improve the drug approval process and expand access. Promising reforms include approval reciprocity with other nations, embracing a wider variety of outcomes-based data, and allowing more medications to be sold over the counter. With the right combination of changes, long-stalled drugs can finally come to market and be easily accessed by patients. "It's long past time for FDA reform. Policymakers need to step up and do right by patients. Millions of Americans will benefit from a faster and more reliable drug approval process." The Taxpayers Protection Alliance (TPA) is a non-profit, non-partisan organization dedicated to educating the public through the research, analysis, and dissemination of information on the government's impact on the economy. SOURCE Taxpayers Protection Alliance 21% more press release views with Request a Demo