PharmExec Blog

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 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

1. Kalydeco and VX-809

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

5. Lambrolizumab

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

13. Serelaxin (RLX030)

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.

Name

Company

Designation

1. Kalydeco and VX-809

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

5. Lambrolizumab

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

13. Serelaxin (RLX030)

Novartis

Acute Heart Failure

14. Drisapersen (GSK2402968/PRO051)

GlaxoSmithKline

Duchenne Muscular Dystrophy

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