MFDS Approves Rare Disease Drugs 'Kamzios Capsules' and 'Wellirek Tablets'

The Ministry of Food and Drug Safety approved four dosages (2.5 mg, 5 mg, 10 mg, 15 mg) of 'Kamzios Capsules (Mavacamten)' by Korea BMS Pharmaceutical, an imported orphan drug used to improve exercise function and symptoms in patients with symptomatic obstructive hypertrophic cardiomyopathy, on the 23rd.

Obstructive hypertrophic cardiomyopathy is a genetic disorder in which the heart muscle thickens, causing abnormal heart function, referring to the condition where the 'left ventricular outflow tract' through which blood is pumped from the heart is obstructed. Kamzios Capsules inhibit cardiac myosin (a protein that induces contraction of heart muscle cells), alleviating excessive contraction of the heart muscle, and are effective in improving exercise function and symptoms in these patients.

Kamzios Capsules are the first domestic treatment for symptomatic obstructive hypertrophic cardiomyopathy. It is expected to provide a new treatment opportunity for patients who previously only received symptomatic therapy to relieve symptoms.

Additionally, the Ministry of Food and Drug Safety approved 'Wellerec Tablets (Belzutifan)' by Korea MSD, a treatment for Von Hippel-Lindau disease characterized by multiple tumors. Von Hippel-Lindau disease is a rare disorder caused by mutations in tumor suppressor genes, resulting in multiple incurable tumors in the kidneys, central nervous system, pancreas, and other organs.

Wellerec Tablets inhibit the cancer-promoting factor (HIF-2α) related to cell proliferation, angiogenesis, and tumor growth, slowing tumor progression and reducing risks associated with repeated tumor resection surgeries. This drug is used to treat renal cell carcinoma, central nervous system hemangioblastomas, and pancreatic neuroendocrine tumors that do not require immediate surgery, providing new treatment options for patients with this rare disease.

This content was produced with the assistance of AI translation services.