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Mar 26, 2026
Precigen reported $3.4 million in first commercial sales of PAPZIMEOS in Q4 2025 following FDA approval for recurrent respiratory papillomatosis. The drug has received standard of care endorsement from physician experts and now covers approximately 90% of insured U.S. lives. The company has $100.4 million in cash and is pursuing European regulatory approval.
Mar 24, 2026
Egetis Therapeutics completed its rolling New Drug Application for Emcitate® (tiratricol) for MCT8 deficiency in the USA on January 29, 2026. The drug received European Commission approval in February 2025 and launched in Germany in May 2025. The company also provided updates on Japanese regulatory progress and the status of its Aladote® candidate for paracetamol overdose.
Mar 23, 2026
The global orphan drugs market is projected to grow from $223.76 billion in 2023 to $486.51 billion by 2032, with North America leading at 38% market share. Over 500 orphan drugs are approved with more than 800 candidates in clinical trials, driven by regulatory incentives and major pharmaceutical company investments in rare disease therapies.
Mar 23, 2026
GSK's antibody-drug conjugate risvutatug rezetecan has received Orphan Drug Designation in Japan for small-cell lung cancer, marking its sixth global regulatory designation. The decision was based on phase I trial data showing durable responses in extensive-stage SCLC patients. The drug is currently in phase III development for relapsed ES-SCLC.
Mar 20, 2026
Aquestive Therapeutics received a Complete Response Letter from the FDA for Anaphylm (dibutepinephrine) sublingual film, citing deficiencies in human factors validation and packaging. The company plans to resubmit as early as Q3 2026.
Mar 16, 2026
Telix Pharmaceuticals has submitted regulatory applications for TLX101-Px, a brain cancer imaging agent, to both the European Medicines Agency and the U.S. Food and Drug Administration, seeking approval for glioma diagnosis and treatment monitoring.
Mar 15, 2026
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Mar 15, 2026
Savara has submitted a Biologics License Application for MOLBREEVI to the FDA with Priority Review status, targeting an August 22, 2026 decision date for treating autoimmune pulmonary alveolar proteinosis.
Mar 12, 2026
BioVersys has dosed the first patient in a Phase 2b trial evaluating alpibectir-ethionamide (AlpE) in combination with first-line TB drugs, following promising Phase 2a results published in the New England Journal of Medicine.
Mar 12, 2026
Brainstorm Cell Therapeutics announced a $1 million private placement at a premium to market price to support its upcoming Phase IIIb trial of NurOwn stem cell therapy for amyotrophic lateral sclerosis.
Mar 11, 2026
The FDA has granted fast track designation to QRX003 for Netherton Syndrome and IBI3003 for relapsed or refractory multiple myeloma, while also accepting regulatory applications for other therapies addressing serious conditions with unmet medical needs.
Mar 12, 2026
Pembrolizumab-based regimens demonstrated significant survival improvements in two separate trials: KEYNOTE-B15 for muscle-invasive bladder cancer and KEYNOTE-B96 for platinum-resistant ovarian cancer, with FDA approval granted for the ovarian cancer indication.
Mar 11, 2026
The FDA is expected to issue draft guidance reducing testing requirements for biosimilar drugs, potentially lowering development costs by USD 20 million. The move comes as the biosimilars market shows strong growth, with major launches in 2025.
Mar 10, 2026
Acurx Pharmaceuticals announced a new clinical trial program for ibezapolstat in patients with recurrent C. difficile infection, building on Phase 2 results showing 96% clinical cure rates with no recurrence while sparing the gut microbiome.
Mar 10, 2026
The US FDA granted priority review and the European Medicines Agency validated applications for Enhertu as post-neoadjuvant treatment for HER2-positive early breast cancer, based on Phase III trial data showing 53% reduction in recurrence risk.
Mar 09, 2026
Regulatory authorities in the US and EU are proposing measures to reduce clinical data requirements and streamline approval processes for biosimilar drugs, aiming to accelerate access to lower-cost alternatives to biologic medications.
Mar 09, 2026
OS Therapies progresses toward Accelerated Approval for OST-HER2 in osteosarcoma, with FDA elevating meeting status and global regulatory submissions on track for Q1 2026.
Mar 09, 2026
ImmunityBio resubmitted its supplemental BLA to the FDA for ANKTIVA in papillary bladder cancer after providing additional data. The company also received encouragement from Saudi regulators to submit a recombinant BCG application and expand ANKTIVA approvals.
Mar 09, 2026
OS Therapies progresses toward Accelerated Approval for OST-HER2 in osteosarcoma, with FDA elevating meeting status and global regulatory submissions on track for Q1 2026.
Mar 09, 2026
FDA elevated OS Therapies' Type D meeting to Type B pre-BLA meeting for OST-HER2 in osteosarcoma, signaling transition to Accelerated Approval discussions. Clinical data submission targeted for end of Q1 2026.
