The week between Nov 16 to 22 2024, marked significant milestones in the biopharma landscape, with approvals, expanded indications, and progress in key therapeutic areas.
Biologics and New Drug Applications (NDAs/BLA)
This week, regulatory bodies, including the FDA and others, granted approvals and designations across multiple therapies, especially in biologics and expanded indications:
New Biologic Approvals
- Marstacimab (Pfizer): Approved by the European Commission (EC) for routine prophylaxis of bleeding episodes in severe hemophilia A and B (Approval Date: Nov 20).
- Zanidatamab (Jazz Pharmaceuticals): FDA-approved for HER2-positive biliary tract cancer (Approval Date: Nov 21).
2 Major Expanded Indications Approved:
- Bimekizumab (UCB): FDA-approved for moderate to severe hidradenitissuppurativa (Approval Date: Nov 20).
- RSV Vaccine (Adjuvanted) (GSK): PMDA approval in Japan for adults aged 50-59 at increased risk of severe RSV disease (Approval Date: Nov 20).
- FDA-Orphan Drug Designation: IMSB-301 (ImmuneSensor Therapeutics) for Aicardi-Goutieres Syndrome (Designation Date: Nov 20).
- Fast-Track Designation: ALE-P02 (Alentis Therapeutics) for advanced squamous cell carcinoma with CLDN1 positivity (Designation Date: Nov 18).
- Breakthrough Therapy Designation:Libevitug (Huahui Health) for Hepatitis D virus infection (Designation Date: Nov 18).
Clinical Trial Progress in Key Therapeutic Areas
5 Oncology Drugs in Active Trials:
- TGN-S11 (Toragen): Presented Phase I data for HPV-associated cancer (Date: Nov 18).
- 64Cu-PSMA I&T (Curium): Completed Phase III patient enrollment for prostate cancer (Date: Nov 19).
- Pembrolizumab (MSD): Phase III positive topline data for metastatic NSCLC (Date: Nov 20).
- Adicet’s Allogeneic Gamma Delta T Cell Therapy: Initiated Phase I trials for ccRCC (Date: Nov 18).
- Anti-NKG2DL CAR-T Cell Therapy (CytoMed): First patient dosed in Phase I trial for hematologic malignancies (Date: Nov 20).
2. Genetic and Metabolic Diseases.
4 Major Trials in Genetic and Metabolic Conditions:
- Icovamenib (Biomea Fusion): Positive Phase II data for Type 1 and Type 2 diabetes (Date: Nov 18).
- Obicetrapib (New Amsterdam): Phase III data in familial hypercholesterolemia (Date: Nov 18).
- NGN-401 (Neurogene): Negative Phase I/II data for Rett syndrome (Date: Nov 18).
- RGX-202 (REGENXBIO): Positive data for Duchenne muscular dystrophy in Phase I/II (Date: Nov 19).
3. Nervous System Diseases
4 Nervous System Drugs in Key Trials:
- Bromophenoxyazole (NMD Pharma): First patient dosed in Phase IIa trial for CMT disease (Date: Nov 18).
- Dalzanemdor (Sage Therapeutics): Presented negative Phase II data for Huntington's-related cognitive impairment (Date: Nov 20).
- AMT-260 (uniQure): Phase I initiation for medial temporal lobe epilepsy (Date: Nov 21).
- Nomlabofusp (Larimar Therapeutics): Positive Phase II data for FXN ataxia (Date: Nov 18).
3 Infectious Disease Therapies in Progress:
- TUNE-401 (Tune Therapeutics): Positive preclinical data for chronic Hepatitis B virus (Date: Nov 19).
- GIGA-2339 (GigaGen): First patient dosed in Phase I for Hepatitis B (Date: Nov 19).
- Umbilical Cord Lining Stem Cell Therapy (Restem): Positive Phase I data for polymyositis/dermatomyositis (Date: Nov 18).
Drug Approval/R&D Trends in China
China continues to lead regulatory and R&D activities:
- BLA Approvals: Bevacizumab biosimilar (Hualan Biological Engineering) for Metastatic colorectal cancer (NMPA Approval Date: Nov 15).
- Expanded Indications: Obicetrapib received approval for hypercholesterolemia (Approval Date: Nov 17).
Key Trends and Takeaways
- Strategic Expanded Indications: Approvals like Bimekizumab demonstrate the value of broadening indications in chronic diseases.
- Focus on Rare Diseases: Orphan and special designations continue to support therapies for rare conditions like Aicardi-Goutieres Syndrome and FXN ataxia.
- Infectious Disease Prevention: Active trials highlight the importance of vaccines and therapies for chronic Hepatitis B and RSV.
Stay tuned for more updates on drug development and approvals. Visit our website to download the full report and explore deeper insights into the evolving global innovative drug R&D landscape.
