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The List So Far FDA Breakthrough Designations
With announcements pouring in since the beginning of this year, FDA’s fourth and newest expedited approval pathway for medicines has begun to bear fruit. Established in 2012 within the FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy designations have so far totaled 25 as of the end of July.
With announcements pouring in since the beginning of this year, FDA’s fourth and newest expedited approval pathway for medicines has begun to bear fruit. Established in 2012 within the FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy designations have so far totaled 25 as of the end of July. With a separate senior management team working solely on the approval of these therapies, FDA stresses the importance of efficient drug development programs that show these medicines demonstrate at least one significant improvement in clinical endpoints for conditions in which therapies already exist. Here’s a list of approved breakthroughs announced thus far in order of their announcement dates:
Name
Company
Designation
Vertex
2 for Cystic Fibrosis
2. Ibrutinib
Pharmacyclics and Janssen Research & Development
3 for relapsed or refractory Mantle Cell Lymphoma, Waldenstrom’s Macroglobulinemia, and Chronic Lymphocytic Leukemia
3. LDK378
Novartis
Condition- ALK+ Non-small cell Lung Cancer
4. Palbociclib
Pfizer
Non-orphan breast cancer
Merck
Melanoma
6. Triple direct-acting antiviral (DAA) treatment regimens of daclatisvir, asunaprevir, and BMS-791325
Bristol-Meyers Squibb
Hepatitis C Virus
7. SD-101
Scioderm
Epidermolysis Bullosa
8. Daratamumab
Genmab/Janssen
Multiple Myeloma
9. DAA Combination of ABT-450/r + ABT-267 + ABT-333 with and without ribavirin
AbbVie
Hepatitis C Virus
10. Obinutuzumab
Genentech
Chronic Lymphocytic Leukemia
11. Sebelipase Alfa
Synageva BioPharma
Early-Onset Lysosomal Acid Lipase Deficiency
12. Asfotase Alfa
Alexion
Perinatal-, Infantile- and Juvenile-Onset Hypophosphatasia
Novartis
Acute Heart Failure
14. Drisapersen (GSK2402968/PRO051)
GlaxoSmithKline
Duchenne Muscular Dystrophy
It appears that this focus on a special pathway is spurring more attention to therapies that represent an advance in the standard of care.
Johnson & Johnson Seeks FDA Approval for Subcutaneous Tremfya Regimen for Ulcerative Colitis
November 22nd 2024Johnson & Johnson has submitted a supplemental Biologics License Application to the FDA for a subcutaneous induction regimen of Tremfya for adults with moderately to severely active ulcerative colitis based on positive Phase III ASTRO trial results.
Key Findings of the NIAGARA and HIMALAYA Trials
November 8th 2024In this episode of the Pharmaceutical Executive podcast, Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses the findings of the NIAGARA trial in bladder cancer and the significance of the five-year overall survival data from the HIMALAYA trial, particularly the long-term efficacy of the STRIDE regimen for unresectable liver cancer.
Fake Weight Loss Drugs: Growing Threat to Consumer Health
October 25th 2024In this episode of the Pharmaceutical Executive podcast, UpScriptHealth's Peter Ax, Founder and CEO, and George Jones, Chief Operations Officer, discuss the issue of counterfeit weight loss drugs, the potential health risks associated with them, increasing access to legitimate weight loss medications and more.
