Category: Regulatory Approval

Regulatory Approval Autoimmune Diseases News

U.S. FDA Approves Amgen’s UPLIZNA® for Generalized Myasthenia Gravis in Adults

amgen

The FDA has approved Amgen’s UPLIZNA for adults with generalized myasthenia gravis (gMG) who are anti-AChR or anti-MuSK antibody positive. UPLIZNA is the first and only CD19-targeted B-cell therapy approved for this indication, offering a convenient dosing schedule of just two maintenance doses per year following the initial loading regimen. The approval is based on strong efficacy data from the Phase 3 MINT trial.

Regulatory Approval Healthcare & Pharmaceuticals News Oncology

U.S. FDA Grants Full Approval to Lilly’s Jaypirca® for Expanded Use in Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma

Eli Lilly and Company

U.S. FDA has granted full approval to Eli Lilly’s Jaypirca (pirtobrutinib) for adults with relapsed or refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). This expanded indication allows the use of the non-covalent BTK inhibitor immediately following prior covalent BTK inhibitor therapy. The decision is bolstered by data from the Phase 3 BRUIN CLL-321 trial, which demonstrated a statistically significant improvement in progression-free survival compared to standard combinatorial therapies.

Regulatory Approval Autoimmune Diseases Healthcare & Pharmaceuticals News

Johnson & Johnson’s Imaavy Wins European Commission Approval for Generalized Myasthenia Gravis

JohnsonAndJohnson

Johnson & Johnson has secured European Commission approval for Imaavy (nipocalimab), a novel treatment for generalized myasthenia gravis (gMG). This authorization marks the first FcRn blocker approved in Europe for a broad population that includes both adults and adolescents (aged 12+), offering a new option for sustained disease control.

Regulatory Approval Healthcare & Pharmaceuticals News

Novartis Receives FDA Approval for Itvisma, Expanding Gene Therapy to Older Children and Adults with SMA

novartis

Novartis has secured FDA approval for Itvisma (onasemnogene abeparvovec-brve), marking a historic milestone as the first gene replacement therapy for spinal muscular atrophy (SMA) available to children aged two and older, teens, and adults. This one-time intrathecal injection offers a “game-changing” new treatment option for patients previously ineligible for gene therapy.

Regulatory Approval Healthcare & Pharmaceuticals News Oncology

U.S. FDA Approves PADCEV® and KEYTRUDA® Combo for Patients with Bladder Cancer

pfizer news

The FDA has granted approval for PADCEV® plus KEYTRUDA® as a perioperative treatment for cisplatin-ineligible patients with muscle-invasive bladder cancer (MIBC). Clinical data reveals the combination reduces the risk of recurrence, progression, or death by 60% compared to surgery alone, marking a major shift in the standard of care.

Regulatory Approval Healthcare & Pharmaceuticals Rare Diseases

U.S. FDA Approves KYGEVVI, the First and Only Treatment for Ultra-Rare Mitochondrial Disease TK2d

UCB News

The U.S. FDA granted approval to KYGEVVI for treating adults and children with Thymidine Kinase 2 Deficiency (TK2d), an ultra-rare, life-threatening mitochondrial disease. Developed by UCB, KYGEVVI is the first and only approved therapy for TK2d, demonstrating a profound 86% reduction in the risk of death and significant improvement in motor function for patients with early-onset disease.

Regulatory Approval Autoimmune Diseases News Regulatory

U.S. FDA Approves Lilly’s Omvoh (mirikizumab-mrkz) as a Single-Injection Monthly Maintenance Dose for Ulcerative Colitis

Eli Lilly and Company

Eli Lilly has received FDA approval for a new single-injection maintenance dose of Omvoh (mirikizumab-mrkz) for adults with moderately to severely active ulcerative colitis. This approval simplifies the treatment regimen from two monthly injections down to just one, offering the same proven efficacy in a more convenient format. The new 200 mg/2 mL prefilled pens and syringes are expected to be available in the U.S. in early 2026.

Regulatory Approval News Oncology

GSK’s Blenrep Gets U.S. FDA Approval for Multiple Myeloma Treatment

GSK - GlaxoSmithKline

The FDA has approved GSK’s Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone for adult patients with relapsed or refractory multiple myeloma. The decision, backed by the pivotal DREAMM-7 trial, introduces a new, accessible anti-BCMA treatment that significantly improved overall survival and progression-free survival compared to the standard of care. This milestone offers renewed hope for patients who have relapsed after at least two prior lines of therapy.

Regulatory Approval News

AstraZeneca’s Tezspire Gains EU Approval for Nasal Polyps, Offering New Hope for Patients

AstraZeneca

The European Commission has officially approved AstraZeneca’s Tezspire (tezepelumab) for adults with severe chronic rhinosinusitis with nasal polyps (CRSwNP). This new add-on therapy offers hope for patients whose condition is uncontrolled by standard treatments, demonstrating efficacy in reducing polyp size, alleviating congestion, and decreasing the need for surgery.