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The FDA has granted a third approval for WELIREG, authorizing its use as a treatment for patients with rare endocrine system tumors known as pheochromocytoma or paraganglioma (PPGL). This approval applies to individuals aged 12 and older with locally advanced, unresectable, or metastatic PPGL, making WELIREG the first oral therapy available for advanced cases of the disease. LAS VEGAS, May 28, 2025 /PRNewswire/ -- Pheochromocytomas and paragangliomas (PCPG) are highly vascular neuroendocrine tumors originating from chromaffin cells of the adrenal medulla or from neural crest-derived cells located outside the adrenal glands. These tumors are categorized based on their location, either in the adrenal medulla or in extra-adrenal paraganglia. They arise from sympathetic tissues found in the adrenal glands or along the abdomen, or from parasympathetic tissues in areas such as the head, neck, and thorax. Sympathetic tumors often secrete large quantities of catecholamines and are found in the adrenal medulla in about 80% of cases. The remaining 20% are extra-adrenal, typically located along the prevertebral and paravertebral sympathetic ganglia in the chest, abdomen, or pelvis. In 2024, there were 4,900 incident PCPG cases across the 7MM, with numbers expected to rise in the coming years. Although significant progress has been made in understanding these tumors, accurately predicting their malignancy remains challenging in the absence of detectable metastases. Surgical resection remains the primary treatment for most localized PCPGs when feasible. In cases of advanced or metastatic disease, management typically involves symptom control using medications such as alpha-blockers, beta-blockers, and inhibitors of catecholamine synthesis to avoid serious complications. When surgery is not viable, treatment options include systemic chemotherapy with Cyclophosphamide, Vincristine, and Dacarbazine (CVD), as well as radiolabeled MIBG therapy ( AZEDRA, which was approved in 2018 but discontinued in early 2024). However, the effectiveness of CVD in improving overall survival is unclear, as most patients eventually face disease progression and death. Learn more about the PCPG treatment @ New Treatment for Pheochromocytomas and Paragangliomas DEMSER (metyrosine) was authorized for use in the United States in 1979 to treat pheochromocytoma. This FDA-approved drug is indicated for preoperative preparation, treatment of patients who are not candidates for surgery, and long-term management of malignant pheochromocytoma. In January 2019, ONO gained approval in Japan to produce and sell DEMSER capsules aimed at controlling catecholamine excess in pheochromocytoma patients. Later, in July 2020, the US FDA approved the first generic version of DEMSER oral capsules, manufactured by Amneal Pharmaceuticals, marking the initial generic approval for DEMSER in the US. In May 2025, Merck announced that the FDA approved WELIREG, the company's oral inhibitor of hypoxia-inducible factor-2 alpha (HIF-2α), for treating adults and children aged 12 and older with locally advanced, inoperable, or metastatic pheochromocytoma or paraganglioma (PPGL). The approval was based on findings from the LITESPARK-015 clinical trial, a single-arm study in which the primary measure of efficacy was the objective response rate (ORR). The trial included 72 patients in a single group (Cohort A1) with measurable disease confirmed by blinded independent central review (BICR) per RECIST v1.1. All participants had a histopathological diagnosis of PPGL and either locally advanced or metastatic disease not suitable for surgical or curative treatment. Their blood pressure was adequately controlled (below 150/90 mm Hg for adults and below 135/85 mm Hg for adolescents) without recent changes in antihypertensive therapy. Patients with carcinomatous meningitis were excluded. Participants received WELIREG at 120 mg once daily until disease progression or the onset of unacceptable toxicity. The primary efficacy endpoint was ORR as assessed by BICR under RECIST v1.1 criteria. Secondary endpoints included duration of response and time to initial response. WELIREG's prescribing information includes a boxed warning noting that exposure during pregnancy may cause harm to the fetus. Healthcare providers are advised to confirm pregnancy status before starting treatment and to counsel patients on the importance of using effective non-hormonal contraception, as WELIREG can reduce the effectiveness of hormonal methods. Additionally, the drug may lead to severe anemia requiring blood transfusions and serious hypoxia, which could necessitate treatment discontinuation, supplemental oxygen, or hospitalization. Regular monitoring for anemia is recommended before and during therapy. Find out more on FDA-approved PCPG drugs @ Pheochromocytomas and Paragangliomas Treatment Options Ongoing clinical trials are assessing a range of targeted therapies aimed at improving treatment outcomes for PCPG. These include radionuclide therapies using SSTR2 agonists and antagonists, alpha-emitting agents, and SSTR2 analogs. Other approaches under investigation involve Dopamine Receptor D2 (DRD2) modulators and ClpP agonists. Each therapy employs distinct mechanisms of action, offering promising advantages and making them key candidates in the advancing PCPG therapeutic landscape. Radionuclide therapy works by delivering radiolabeled peptides to tumor cells via specific receptor targeting. Current clinical guidelines suggest that radioligand therapy may serve as a suitable first-line treatment for patients with slowly to moderately progressive metastatic PCPG, provided the tumor demonstrates uptake of the relevant radioisotope. Several prominent institutions, including the National Cancer Institute (NCI), M.D. Anderson Cancer Center, Gustave Roussy Cancer Campus, and Columbia University are actively conducting clinical studies in this area. Discover which therapies are expected to grab major PCPG market share @ Pheochromocytomas and Paragangliomas Market Report Some of the drugs in the pipeline include ONC201 (Jazz Pharmaceuticals/Chimerix), [212Pb]VMT-α-NET (Perspective Therapeutics), and LUTATHERA (Lutetium [177Lu] oxodotreotide/dotatate) (Novartis), among others. ONC201, also known as dordaviprone, is a first-in-class small-molecule imipridone that specifically targets the G-protein-coupled receptor DRD2 and the mitochondrial protease ClpP. It was investigated in a Phase II, open-label, investigator-initiated trial at the Cleveland Clinic, involving 30 patients with rare neuroendocrine tumors. Interim results demonstrated a 50% overall response rate (ORR) in paraganglioma cases with dopamine-secreting tumors located outside the brain. These findings, based on investigator assessments, were presented at the 2021 ASCO Annual Meeting and later published in Clinical Cancer Research in 2022. [212Pb] VMT-α-NET is a Targeted Alpha Therapy (TAT) radiopharmaceutical in clinical development for diagnosing and treating SSTR2-positive neuroendocrine tumors, a challenging and uncommon cancer type. Perspective Therapeutics is conducting a multicenter, open-label Phase I/IIa dose-escalation and expansion trial (NCT05636618) in patients with unresectable or metastatic SSTR2-expressing tumors who have not previously received peptide receptor radionuclide therapy. The FDA has granted Fast Track Designation (FTD) for this program based on encouraging preclinical data, regardless of patients' prior treatment history. As of February 2025, 30 patients have been enrolled in Cohort 2, with regulatory alignment pending for Cohort 3. In April 2025, Perspective Therapeutics announced that data from both the [212Pb] VMT-α-NET and [212Pb] VMT01 programs had been accepted for presentation at the upcoming ASCO Annual Meeting, scheduled for May 30–June 3, 2025, in Chicago, Illinois. Details for regular abstracts are expected to be released on May 22, 2025, at 5:00 PM EDT. Discover more about drugs for PCPG in development @ Pheochromocytomas and Paragangliomas Clinical Trials The anticipated launch of these emerging therapies for PCPG are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the PCPG market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for PCPG is expected to grow from USD 310 million in the 7MM in 2024 at a significant CAGR by 2034. Increasing awareness and improved detection rates are contributing to a rising patient pool. Additionally, ongoing research and development of novel treatment options, including precision medicine approaches, are fueling market expansion. The growing prevalence of these rare neuroendocrine tumors, coupled with supportive healthcare infrastructure, further boosts market opportunities worldwide. DelveInsight's latest published market report, titled as Pheochromocytomas and Paragangliomas Market Insight, Epidemiology, and Market Forecast – 2034 , will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the PCPG country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The PCPG market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Incident Cases of PCPG Occurrence or Absence of mutation in PCPG Age-specific Cases of PCPG Stage-specific Cases of PCPG The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM PCPG market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this PCPG market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the PCPG market. Also, stay abreast of the mitigating factors to improve your market position in the PCPG therapeutic space. 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