Latest news with #Welireg
Medscape
15-05-2025
- Health
- Medscape
FDA Okays Belzutifan for Pheochromocytoma or Paraganglioma
The US Food and Drug Administration (FDA) has approved belzutifan (Welireg, Merck) for adult and pediatric patients aged 12 years or older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). It is the first US approval of 'an oral therapy for PPGL,' the FDA said in a press release. The hypoxia-inducible factor 2 alpha inhibitor was previously approved for von Hippel-Lindau disease and advanced renal cell carcinoma. PPGLs are rare and related neuroendocrine tumors that can secrete catecholamines. Pheochromocytoma develops in the adrenal glands, while paraganglioma can develop in various other places. The tumors can lead to life-threatening hypertensive crises. Approval was based on the open-label, multicohort LITESPARK-015 trial with 72 patients having PPGL tumors not amenable to surgery or curative treatment. The objective response rate was 26%; median duration of response was 20.4 months (95% CI, 8.3, NR). Of the 60 patients on baseline antihypertensive medications, 19 (32%) had a reduction in at least one antihypertensive medication by at least 50% for at least 6 months. The most common adverse reactions in 25% or more of subjects included anemia, fatigue, musculoskeletal pain, decreased lymphocytes, increased alanine aminotransferase, increased aspartate aminotransferase, increased calcium, dyspnea, increased potassium, decreased leukocytes, headache, increased alkaline phosphatase, dizziness, and nausea. Labelling carries a black box warning of embryo-fetal toxicity. The recommended dose for adults is 120 mg oral belzutifan, taken once daily. The recommended dose for pediatric patients aged 12 years and older is based on body weight. For those with a body weight of 40 kg or more, the dose is 120 mg oral belzutifan, taken once daily. For pediatric patients under 40 kg, the dose is 80 mg oral belzutifan, taken once daily. Treatment continues until disease progression or unacceptable toxicity. Ninety 40 mg tablets cost $32,624.23, according to
Yahoo
14-05-2025
- Business
- Yahoo
FDA Approves Merck's Kidney Cancer Drug For Rare Type Of Neuroendocrine Tumors
The U.S. Food and Drug Administration (FDA) on Wednesday approved Merck & Co. Inc.'s (NYSE:MRK) Welireg (belzutifan) for adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). This represents the first FDA approval of an oral therapy for PPGL, rare neuroendocrine tumors that can spread to other parts of the body. Welireg received approval from the FDA and European Medicines Agency for adult patients with advanced renal cell carcinoma (RCC).Efficacy was evaluated in LITESPARK-015, an open-label, multi-cohort trial. The major efficacy outcome measure was objective response rate (ORR). Additional efficacy outcome measures were duration of response (DOR) and the number of patients who had a reduction in at least one antihypertensive medication by at least 50%, maintained for at least six months. ORR was 26%. Median DOR was 20.4 months. Of the 60 patients on baseline antihypertensive medications, 19 (32%) had a reduction in at least one antihypertensive medication by at least 50% for at least six months. The recommended belzutifan dose for adults is 120 mg administered orally once daily. The recommended dose for pediatric patients 12 years and older is based on body weight. For pediatric patients weighing ≥ 40 kg, the dose is 120 mg orally once daily. For pediatric patients weighing < 40 kg, the dose is 80 mg orally once daily. In the first quarter of 2025, Welireg generated sales of $137 million, 62% year over year. Price Action: MRK stock is down 3.45% at $73.98 at the last check on Wednesday. Read Next:Photo by tada images via Shutterstock UNLOCKED: 5 NEW TRADES EVERY WEEK. Click now to get top trade ideas daily, plus unlimited access to cutting-edge tools and strategies to gain an edge in the markets. Get the latest stock analysis from Benzinga? MERCK & CO (MRK): Free Stock Analysis Report This article FDA Approves Merck's Kidney Cancer Drug For Rare Type Of Neuroendocrine Tumors originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Reuters
14-05-2025
- Health
- Reuters
US FDA expands use of Merck's cancer drug for adrenal gland tumors
May 14 (Reuters) - The U.S. Food and Drug Administration said on Wednesday it has approved the expanded use of Merck's (MRK.N), opens new tab cancer drug to treat two types adrenal gland tumors. The drug, Welireg, became the first FDA-approved oral treatment for the two tumors — pheochromocytoma and paraganglioma. The regulator's approval was based on a study in which Welireg showed an objective response rate of 20.6%. ORR is the percentage of people who have a partial response or complete response to the treatment within a certain period of time.
Yahoo
14-05-2025
- Health
- Yahoo
US FDA expands use of Merck's cancer drug for adrenal gland tumors
(Reuters) -The U.S. Food and Drug Administration said on Wednesday it has approved the expanded use of Merck's cancer drug to treat two types adrenal gland tumors. The drug, Welireg, became the first FDA-approved oral treatment for the two tumors — pheochromocytoma and paraganglioma. The regulator's approval was based on a study in which Welireg showed an objective response rate of 20.6%. ORR is the percentage of people who have a partial response or complete response to the treatment within a certain period of time.
Yahoo
27-01-2025
- Business
- Yahoo
MSD wins FDA priority review for Welireg in rare endocrine cancer
The US Food and Drug Administration (FDA) will soon decide on whether to expand the label of MSD's Welireg (belzutifan) to include its use for the treatment of the rare cancer pheochromocytoma and paraganglioma (PPGL). The regulatory agency will review MSD's new drug application (NDA) for Welireg under a Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of 26 May 2025. The new label is intended to cover adult and paediatric patients aged 12 years and older with advanced, unresectable, or metastatic PPGL. MSD's submission is based on efficacy and safety data from the Phase II LITESPARK-015 trial (NCT04924075), which evaluated Welireg in patients with advanced PPGL. The study measured the objective response rate (ORR) and duration of response (DOR) as primary endpoints and included other secondary endpoints such as disease control, progression-free survival (PFS), and safety. Results from the trial are expected to be presented at an upcoming medical meeting. MSD acquired Welireg when it bought Peloton Therapeutics for an upfront payment of $1.05bn in 2019. In August 2021, the drug was first approved by the FDA for the treatment of a rare disorder called von Hippel-Lindau (VHL) disease. Welireg is also approved for treating some patients with advanced renal cell carcinoma (RCC). PPGL is a group of rare neuroendocrine tumours originating from adrenal gland tissue or paraganglia. These tumours are often challenging to treat in advanced stages, and current therapeutic options are limited. A gap in the PPGL treatment market was left following the withdrawal of Lantheus's Azedra (iobenguane I-131), a radiopharmaceutical therapy approved by the FDA for treating unresectable, locally advanced, or metastatic PPGL requiring systemic anticancer therapy in 2018. Azedra was the first FDA-approved therapy specifically for this rare indication. However, the drug was voluntarily withdrawn from the market due to manufacturing challenges that affected its commercial availability in 2023. Welireg inhibits hypoxia-inducible factor-2 alpha (HIF-2α), thereby targeting pathways involved in tumour growth and survival under low-oxygen conditions. Welireg generated $218m in global sales in 2023, as per MSD's financials, with expectations to reach up to $1.5bn in annual sales in 2030, according to GlobalData's Pharma Intelligence Center. GlobalData is the parent company of Pharmaceutical Technology. Welireg comes with a boxed warning about its potential to cause embryo-foetal harm if used during pregnancy. It can also render hormonal contraceptives ineffective and cause severe anaemia and severe hypoxia. "MSD wins FDA priority review for Welireg in rare endocrine cancer" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
