Luxturna (generic name: voretigene neparvovec-rzyl) is the first gene therapy approved by the U.S. Food and Drug Administration (FDA) to directly target a specific mutation in a gene for the treatment of hereditary retinal dystrophy…
In exciting news for the healthcare sector, an innovative drug called Lumizyme is becoming the focus of attention for patients with rare diseases and their families. The drug has shown great potential for the treatment…
Quviviq (dazodalant) is an innovative insomnia treatment that is unique in its novel mechanism of action. Unlike traditional sleep aids, Quviviq does not induce sleep by depressing the central nervous system, but rather by precisely…
Folotyn (generic name: Pralatrexate), the first drug approved by the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL), has made a number of important advances worldwide…
March 20, 2025 – A breakthrough in cancer treatment has emerged with the approval and introduction of Rezivertinib Mesylate Capsules, a third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) designed to target EGFR-mutated…
May 6, 2020- The U.S. Food and Drug Administration (FDA) today approved Tabrecta (capmatinib) for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) harboring a jump mutation in exon 14 of…
Brineura® (cerliponase alfa), the only FDA-approved enzyme replacement therapy for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease). continues to demonstrate significant clinical benefits in slowing disease progression, even as access barriers persist for families dependent…
Parsabiv (Etelcalcetide), an innovative calcimimetic, was recently formally approved for the treatment of secondary hyperparathyroidism (SHPT) in patients with chronic kidney disease (CKD) receiving dialysis. This approval provides a new treatment option for millions of…
August 5, 2023 – Eli Lilly and Company (Eli Lilly) announced that its RET inhibitor Retevmo (Selpercatinib) has achieved positive top-line data in a Phase III clinical trial for the treatment of patients with RET…
Cushing’s syndrome is a rare disease caused by the overproduction of cortisol from the adrenal glands, and patients often exhibit symptoms such as obesity, hypertension, and diabetes. In recent years, important progress has been made…
March 29, 2025 – Horizon Therapeutics, the manufacturer of Actimmune® (interferon gamma-1b), has announced that the U.S. Food and Drug Administration (FDA) has granted expanded approval for the treatment of severe malignant osteopetrosis in pediatric patients. This decision follows…
Pfizer Inc. recently announced that its third-generation anaplastic lymphoma kinase (ALK) inhibitor Lorviqua (generic name: Lorlatinib) has been approved by the European Commission As a monotherapy for the first-line treatment of adult patients with ALK-positive…
