Wednesday, October 30, 2013

Breakthrough Therapy Designation

What is a Breakthrough Therapy?
A new drug may be designated as a breakthrough therapy by the Food and Drug Administration (FDA) if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Once the breakthrough therapy designation is requested by the drug sponsor, the FDA and sponsor work together to determine the most efficient path forward.
 
Breakthrough Designations Announced By Companies (as of October 2013):
  • Kalydeco - (Vertex) - 2 cystic fibrosis designations
    • in combination with VX-809 in patients with two copies of the F508del mutation 
    • ​monotherapy for other CFTR mutations
  • Ibrutinib - (J&J/Pharmacyclics) - 3 designations
    • mantle cell lymphoma
    • waldenstrom macroglobulinemia (WM)
    • chronic lymphocytic leukemia
  • LDK378 - (Novartis) - ALK+ NSCLC resistant to crizotinib
  • Palbociclib - (Pfizer) - treatment of post-menopausal patients with ER+, HER2- locally advanced or metastatic breast cancer
  • Lambrolizumab - (Merck) - inoperable and metastatic melanoma 
  • Daclatasvir*- (BMS) - interferon-free treatmemt of Hepatitis C *combination of daclatasvir with two other direct-acting antivirals, asunaprevir, an NS3 protease inhibitor and BMS-791325, an NS5B non-nucleoside polymerase inhibitor
  • Daratumumab - (Janssen) - for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD), or who are double refractory to a PI and IMiD
  • SD101 - (Scioderm) - topical cream for the treatment of inherited Epidermolysis Bullosa (EB)
  • ABT-450* - (AbbVie) - interferon-free 3-DAA treatment of Hepatitis C tested both with and without ribavirin *combination of ABT-450 with two other drugs, ABT-267, an NSA5 inhibitor, and ABT-333, a non-nucleoside polymerase inhibitor
  • Obinutuzumab - (Genetech) - for the treatment of chronic lymphocytic leukemia (CLL)
  • Sebelipase Alfa - (Synageva) - for the treatment of early onset lysosomal acid lipase deficiency (LAL deficiency)
  • Asfotase Alfa - (Alexion Pharmaceutical) - for the treatment of patients with hypophosphatasia (HPP) whose first signs or symptoms occurred prior to 18 years of age, including perinatal-, infantile-, and juvenile-onset forms of the disease
  • Serelaxin - (Novartis) - for the treatment of acute heart failure
  • Drisapersen - (GlaxoSmithKline) - for the treatment of Duchenne muscular dystrophy (DMD) in ambulant and non-ambulant boys who carry dystrophin gene mutations amenable to exon 51 skipping
  • BYM338 (bimagrumab) - (Novartis) - for the treatment of sporadic inclusion body myositis (sIBM)
  • Sofosbuvir/ledipasvir combination - (Gilead) - for the treatment of Hepatitis C
  • Firdapse (amifampridine phosphate) - (Catalyst Pharmaceutical) - for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) 
  • Entinostat - (Syndax Pharmaceuticals) - in combination with exemestane for the treatment of metastatic ER-positive breast cancer in postmenopausal women who have progressed on hormonal therapy
  • Arzerra (ofatumumab) - (Genmab A/S, GlaxoSmithKline) - in combination with chlorambucil for the treatment of previously untreated chronic lymphocitic leukemia (CLL) in patients for whom fludarabine-based therapy is inappropriate 
  • Volasertib - (Boehringer Ingelheim) - for the treatment of previously untreated acute myeloid leukemia (AML) in patients over the age of 65
  • Alectinib - (Roche) - for the treatment of non-small cell lung cancer 
  • MK-5172/MK-8742 - (Merck) - for the treatment of Hepatitis C
  • cPMP - (Alexion) - for the treatment of molybdenum cofactor deficiency (MoCD) type A
*As of September 30, 2013, FDA lists 92 total requests for Breakthrough designation, 27 requests granted, and 41 requests denied. FDA does not disclose information regarding specific drugs or sponsors. Currently, about one granted requests has not been publicly announced by their sponsors.


source: FOCR

at