Miplyffa (arimoclomol) / LadRx, Orphazyme, Zevra Therapeutics, Ligand - LARVOL DELTA

Zevra Therapeutics, Inc…announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion regarding the Marketing Authorization Application (MAA) for arimoclomol for the treatment of Niemann-Pick disease type C (NPC), a rare, progressive and life-threatening neurodegenerative disorder. (The Manila Times)

CHMP • Genetic Disorders

Ligand Completes Acquisition of XOMA Royalty, Creating a Portfolio of More than 200 Biopharmaceutical Royalty Assets (The Manila Times) - Acquisition adds seven commercial products including VABYSMO (faricimab-svoa), OJEMDA (tovorafenib), and MIPLYFFA (arimoclomol), andmore than 100 development and commercial-stage assets, creating long-term opportunities to drive growth...Additionally, it includes 14 late-stage development programs, featuring Takeda’s mezagitamab and certain assets from Takeda’s externalized asset portfolio, such as osavampator, volixibat, and OHB-607, along with more than 100 assets in various stages of development....Ligand will provide an updated 5-year outlook during the Company’s Investor Day on December 8, 2026."

M&A • Bronchopulmonary Dysplasia • Diabetic Macular Edema • Diabetic Retinopathy • IgA Nephropathy • Immune Thrombocytopenic Purpura • Immunology • Low Grade Glioma • Lysosomal Storage Diseases • Metabolic Dysfunction-Associated Steatohepatitis • Primary Biliary Cholangitis • Wet Age-related Macular Degeneration

ORF01 - Central Mechanisms Regulating Growth and Neuroendocrine Function (ENDO 2026) - No abstract available

IB-OR01-1.5: Oral Presentations - Molecular and Therapeutic Strategies for Diabetes (ADA 2026) - No abstract available

Diabetes • Metabolic Disorders

CT-OR01-1.5: Oral Presentations - Preventing Type 1 Diabetes (ADA 2026) - No abstract available

Diabetes • Metabolic Disorders • Type 1 Diabetes Mellitus

IP-OR01-1.5: Oral Presentations - Preserving Lean Mass with GLP-1R Therapy: Preclinical Obesity Studies (ADA 2026) - No abstract available

Preclinical • Metabolic Disorders • Obesity

IA-OR01-1.5: Oral Presentations - Molecular Transducers of Insulin Action and Insulin Resistance (ADA 2026) - No abstract available

Metabolic Disorders

AC-OR01-1.5: Oral Presentations - Hypoglycemia across Contexts: Risk, Recognition, and Real-World Safety (With ADA Presidents' Select Abstract Presentation) (ADA 2026) - No abstract available

Clinical • Real-world • Real-world evidence • Hypoglycemia • Metabolic Disorders

EG-OR01-1.5: Oral Presentations - From Plate to Pancreas: Mechanisms Linking Diet to Diabetes (With ADA Presidents' Select Abstract Presentation) (ADA 2026) - No abstract available

Diabetes • Metabolic Disorders

BM-OR01-1.5: Oral Presentations - Empowering Populations: Diabetes Self-Management Education in Practice (ADA 2026) - No abstract available

Clinical • Diabetes • Metabolic Disorders

MIPLYFFA (arimoclomol) Highlights (GlobeNewswire) - "Global Expanded Access Program (EAP): As of March 31, 2026, 122 patients were enrolled in the global EAP...MIPLYFFA was included in the newly updated Clinical Practice Guidelines for the treatment and management of NPC."

Clinical • Clinical guideline • Genetic Disorders

EU: A Marketing Authorisation Application for the evaluation of arimoclomol for the treatment of NPC is under review by the European Medicines Agency (EMA) (GlobeNewswire) - "The Company submitted its response to the EMA’s 120-day list of questions...Arimoclomol has been designated an Orphan Medicinal Product by the EMA."

EMA filing • Orphan drug • Genetic Disorders

Zevra Reports First Quarter 2026 Financial Results and Corporate Update (GlobeNewswire) - "Q1 2026 net revenue of $36.2 million...which includes $24.6 million of MIPLYFFA net revenue...Received nine MIPLYFFA prescription enrollment forms for Niemann-Pick disease type C (NPC) during Q1 2026, bringing the total to 170 since product launch."

Sales • Genetic Disorders

Zevra Reports Third Quarter 2025 Financial Results and Corporate Update (The Manila Times) - "MIPLYFFA: Eight prescription enrollment forms during Q3 2025, bringing the total since product launch to 137; OLPRUVA: One prescription enrollment form during Q3 2025, bringing the total since product launch to 30...$26.1 million for Q3 2025, which includes $22.4 million of MIPLYFFA net revenue, $0.1 million of OLPRUVA net revenue, $2.4 million in net reimbursements from the French EAP for arimoclomol..."

Commercial • Genetic Disorders

OR01-1. Ambulatory Anesthesia - Oral Presentations (0 Credit) (ASA 2025) - No abstract available

Anesthesia

OR01 Oral abstracts: Maternal fetal medicine (FIGO 2025) - No abstract available

Q2 2025 Financial Highlights (Yahoo Finance) - "Revenue, Net: $25.9 million for Q2 2025, which includes $21.5 million of MIPLYFFA net revenue, $0.3 million of OLPRUVA net revenue, $2.6 million in net reimbursements from the French EAP for arimoclomol, $1.2 million in royalties and other reimbursements under the AZSTARYS license agreement, and an upfront payment of $0.3 million from the out-license of our dextrorphan prodrug. For Q2 2024, total net revenue was $4.4 million."

Commercial • CNS Disorders • Genetic Disorders

Zevra Therapeutics Submits Marketing Authorization Application to European Medicines Agency to Review Arimoclomol for the Treatment of Niemann-Pick Disease Type C (The Manila Times) - "Zevra Therapeutics, Inc...announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC)...The EMA will review the application under the centralized marketing authorization procedure...'The extensive data generated for arimoclomol has shown long-term, meaningful clinical outcomes with 5 to 7 years of patient experience across more than 270 NPC patients through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date.'"

EMA filing • Genetic Disorders

OR01. NEUROENDOCRINOLOGY AND PITUITARY: Mechanisms and Gene Changes (ENDO 2025) - No abstract available

Endocrine Disorders

Game Changers: Blockbuster Small-Molecule Drugs Approved by the FDA in 2024. (PubMed, Pharmaceuticals (Basel)) - "Notably, eight of these drugs (including Rezdiffra®, Voydeya®, Iqirvo®, Voranigo®, Livdelzi®, Miplyffa®, Revuforj®, and Crenessity®) are classified as "first-in-class" and have received breakthrough therapy designation. These agents not only exhibit distinct mechanisms of action but also offer substantial improvements in efficacy for patients compared to prior therapeutic options. This article offers a comprehensive analysis of the mechanisms of action, clinical trials, drug design, and synthetic methodologies related to representative drugs, aiming to provide crucial insights for future pharmaceutical development."

FDA event • Journal • Review • Alopecia • Brain Cancer • Breast Cancer • Cardiovascular • Chronic Kidney Disease • Chronic Obstructive Pulmonary Disease • CNS Disorders • Congenital Adrenal Hyperplasia • Cystic Fibrosis • Dermatology • Duchenne Muscular Dystrophy • Endocrine Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Glioma • Hematological Disorders • Hematological Malignancies • Hepatology • Hypertension • Immunology • Infectious Disease • Leukemia • Lung Cancer • Lysosomal Storage Diseases • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis • Muscular Dystrophy • Nephrology • Non Small Cell Lung Cancer • Oncology • Primary Biliary Cholangitis • Psychiatry • Pulmonary Disease • Renal Disease • Respiratory Diseases • Schizophrenia • Solid Tumor • Ventricular Tachycardia

Zevra Announces Publication of MIPLYFFA Mechanism of Action Manuscript in Molecular Genetics and Metabolism (GlobeNewswire) - "The publication presents data demonstrating that arimoclomol enters the cell and increases the translocation of translation factors EB and E3 (FTEB & TFE3) from the cytosol to the nucleus, a key initial step for triggering a cascade of downstream events. Specifically, TFEB & TFE3 upregulate the coordinated lysosomal expression and regulation (CLEAR) genes including NPC1, which is essential for the regulation of lysosomal function. Increased CLEAR gene expression then causes higher NPC1 protein levels in the lysosomes, leading to greater correction of aberrant cholesterol trafficking. Of note, in animal NPC models, greater correction of aberrant cholesterol trafficking was shown to correlate with the improvement of specific neurological behaviors, such as rearing and gait."

Preclinical • Genetic Disorders • Lysosomal Storage Diseases • Rare Diseases

Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease. (PubMed, Mol Genet Metab) - "Arimoclomol, an orally available small molecule, is the first FDA-approved treatment for NPC when used in combination with miglustat...By also amplifying expression of CLEAR genes associated with autophagy, arimoclomol has the potential to act on different pathways and improve cell viability independent of NPC1 protein levels and functionality. In summary, the findings presented illustrate how arimoclomol improves lysosomal function and potentially autophagy flux to decrease lipid burden in NPC patient fibroblasts."

Journal • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • TFE3 • TFEB

Zevra Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $150 Million (GlobeNewswire) - "Zevra Therapeutics, Inc...announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million. 'We have reached another key milestone with the closing on the sale of our PRV, which we received following the successful approval of MIPLYFFA (arimoclomol) from the U.S. Food and Drug Administration (FDA)'...'The $150 million in gross proceeds is non-dilutive capital that further enhances our financial flexibility to support our strategic priorities, which includes executing on the commercial launches of MIPLYFFA and OLPRUVA, supporting our ongoing Phase 3 trial for celiprolol, and augmenting our solid balance sheet to support future investments that are in alignment with our strategic plan'."

Financing • CNS Disorders • Metabolic Disorders

FDA approval of Miplyffa and Aqneursa: A dual breakthrough for the treatment of Neimann-Pick disease type C. (PubMed, Alzheimers Dement (N Y)) - No abstract available

FDA event • Journal • Frontotemporal Lobar Degeneration

Zevra Therapeutics Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million (GlobeNewswire) - "Zevra Therapeutics, Inc...today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million upon the closing of the transaction, which is expected to take place within 30 to 45 days, subject to customary closing conditions...'This non-dilutive capital strengthens our balance sheet by adding gross cash proceeds of $150 million, supporting continued investment in our strategic priorities, which include executing the commercial launches of MIPLYFFA and OLPRUVA, and advancing our pipeline of product candidates to address unmet needs within the rare disease community.'"

Financing • CNS Disorders • Metabolic Disorders