The Berke Report: Clinical and Commercial Updates
Updated: Jul 12
Commercial:
FDA grants accelerated approval to Sarepta’s Duchenne muscular dystrophy gene therapy. The disease occurs in up to one in every 5,000 newborn males worldwide (pmlive)
FDA Approves Pfizer’s LITFULO™ (Ritlecitinib) for Adults and Adolescents With Severe Alopecia Areata, LITFULO is the first and only treatment for severe alopecia areata approved for patients as young as 12 (PR)
FDA approves Argenx's drug for muscle-weakening disease. The drug, branded as Vyvgart Hytrulo, is expected to be available in the U.S. next month and will come at a list price of $15,773, the company told Reuters. (reuters)
Pfizer’s TALZENNA® in Combination with XTANDI® Receives U.S. FDA Approval. TALZENNA is first and only PARP inhibitor approved for use with an existing standard of care (XTANDI) for adult patients with homologous recombination repair (HRR) gene-mutated metastatic castration resistant prostate cancer (PR)
FDA approves Eli Lilly-Boehringer's diabetes drugs for children. The approvals were based on a study that showed patients aged between 10 years and 17 years recorded lower average blood sugar compared with placebo when treated with empagliflozin - the active ingredient in the two drugs (reuters)
Zealand Pharma Submits Marketing Authorization Application for Dasiglucagon for Treatment of Severe Hypoglycemia in Diabetes to the European Medicines Agency (PR)
ARS Pharmaceuticals Announces PDUFA Date Extension for neffy® for the Treatment of Allergic Reactions, Including Anaphylaxis (biospace)
CAMZYOS is the first and only FDA-approved cardiac myosin inhibitor that specifically targets the underlying source of obstructive HCM. VALOR-HCM is the second Phase 3 trial in which CAMZYOS demonstrated significant improvement in symptoms of obstructive HCM (PR)
Roche has secured approval for Columvi, a treatment for certain people with diffuse large B-cell lymphoma. Columvi is one of a new class of emerging therapies, antibodies which contain two antigen-binding sites and which can therefore simultaneously target two different epitopes (thepharmaletter)
U.S. FDA Approves Addition of Positive Data from Phase 3 VALOR-HCM Study to Bristol Myers’s CAMZYOS® Label (PR)
Braeburn's BRIXADI™ (buprenorphine) Extended-Release Subcutaneous Injection Receives FDA Approval for Moderate to Severe Opioid Use Disorder (PR)
Clinical:
Synlogic Announces Initiation of Synpheny-3 Global, Pivotal Phase 3 Study Evaluating SYNB1934 for Treatment of Phenylketonuria (PR)
FDA Advisory Committee Votes Unanimously to Confirm the Clinical Benefit of LEQEMBI® (lecanemab-irmb) for the Treatment of Alzheimer’s Disease (PR)
Praxis Precision Medicines Announces Plans to Begin Ulixacaltamide Phase 3 in Essential Tremor by Year End After Completing End of Phase 2 Meeting with FDA (PR)
Rain Oncology Announces Topline Results from Phase 3 MANTRA Trial of Milademetan for the Treatment of Dedifferentiated Liposarcoma (PR)
Galecto Completes Dosing in 52-Week Phase 2b GALACTIC-1 Trial of GB0139 in Idiopathic Pulmonary Fibrosis (PR)
Biomea Fusion (BMEA) Presents Positive Clinical Data from Initial Cohorts of Ongoing Phase II Study of BMF-219 in Patients with Type 2 Diabetes Mellitus (streetinsider)
MAIA Biotechnology Announces Updates in Enrollment in Phase II Clinical Trial: THIO-101 Has Enrolled 29 Patients (PR)
Rallybio Announces Proof-of-Concept Results and Development Updates for RLYB212, a Novel Monoclonal anti-HPA-1a Antibody to Prevent Fetal and Neonatal Alloimmune Thrombocytopenia (PR)
Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial (medtechalert)
Another of Mersana's ADCs slapped with FDA hold, this time over bleeding events that include 5 deaths (fiercebiotech)
Designations:
Avenge Bio Receives FDA Orphan Drug Designation for AVB-001 for the Treatment of Mesothelioma (PR)
Cidara Therapeutics Receives U.S. FDA Fast Track Designation for CD388, a Novel Drug-Fc Conjugate Targeting Influenza A and B (PR)
Aardvark Therapeutics Announces FDA Orphan Drug Designation Granted to ARD-101, a Novel Drug Candidate for Prader-Willi Syndrome (PR)
Teikoku Pharma granted FDA Fast Track Designation for TPU-006, a Novel 4-day Dexmedetomidine Transdermal System for Post Operative Pain Management (PR)
AceLink Therapeutics Receives FDA Clearance to Initiate a Phase 2 Study of AL1211 in Patients with Fabry Disease (PR)
Milvexian Granted U.S. FDA Fast Track Designation for All Three Indications Under Evaluation in Phase 3 Librexia Program: Ischemic Stroke, Acute Coronary Syndrome and Atrial Fibrillation (PR)
Takeda and HUTCHMED Announce New Drug Application (NDA) for Fruquintinib for Treatment of Previously Treated Metastatic Colorectal Cancer Granted Priority Review (PR)
Aardvark Therapeutics Announces FDA Orphan Drug Designation Granted to ARD-101, a Novel Drug Candidate for Prader-Willi Syndrome (PR)