Como miembros de NORD, tenemos acceso al listado de los medicamentos huérfanos recientemente aprobados por la FDA de Estados Unidos. El listado incluye, entre otras cosas, medicamentos y dispositivos aprobados, designaciones de drogas huérfanas, designaciones de terapias innovadoras y designaciones de vía rápida.

Transcribimos literalmente (sin traducción, porque no queremos arriesgarnos a una mala traducción técnica) dicho listado del boletín-e de NORD. Disculpen los inconvenientes.


FDA News

Recent Drug and Device Approvals

  • Farydak (panobinostat) has been approved for the treatment of patients with multiple myeloma.  This is the first histone deacetylase (HDAC) inhibitor approved to treat multiple myeloma and is intended for patients who have received at least two prior standard therapies. Farydak is marketed by Novartis Pharmaceuticals.
  • Cresemba (isavuconazonium sulfate) has been approved to treat adults with the rare, serious, fungal infections invasive aspergillosis and invasive mucormycosis.  Cresemba is marketed by Astellas Pharma US, Inc.
  • Zarxio (filgrastim-sndz) has been approved as the first biosimilar product in the US.  Zarxio is biosimilar to Amgen Inc.’s Neupogen (filgrastim) and is approved for the same indications, to help prevent infections in cancer patients receiving chemotherapy.  Zarxio is manufactured by Sandoz, a Novartis company.
  • Lixelle Beta 2-microglobulin apheresis column has been approved as the first device to treat dialysis-related amyloidosis.  The Lixelle column is manufactured by Kaneka Corporation and distributed in the U.S. by Kaneka Pharma America.
  • Unituxin (dinutuximab) has been approved as the first therapy for pediatric patients with high-risk neuroblastoma.  This is the second drug approval granted through FDA’s rare pediatric disease review voucher program.  Unituxin is marketed by United Therapeutics.
  • Cholbam (cholic acid) has been approved as the first treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders). This is the third drug approval granted through FDA’s rare pediatric disease review voucher program. Cholbam is marketed by Asklepion Pharmaceuticals LLC.
  • Anthrasil, anthrax immune globulin intravenous (human) has been approved to treat patients with inhalational anthrax in combination with appropriate antibacterial drugs. The product is manufactured by Cangene Corporation.

Recent Orphan Drug Designations

  • Portola Pharmaceuticals for andexanet alfa for the treatment of bleeding associated with Factor Xa inhibitors
  • Oncolytics Biotech for pelareorep for the treatment of ovarian, fallopian tube and primary peritoneal cancers
  • NightstaRx Ltd for a gene therapy to treat choroideremia
  • Cell Medica for CMD-003, an immunotherapy to treat Epstein-Barr virus positive non-Hodgkin lymphomas
  • Selten Pharma for tacrolimus (SPI-026), an investigational bone morphogenetic protein receptor type II pathway activator for pulmonary arterial hypertension
  • Aeolus Pharmaceuticals, Inc. for AEOL 10150 for treatment of idiopathic pulmonary fibrosis
  • AB Science SA for masitinib in the treatment of amyotrophic lateral sclerosis
  • Dilaforette for sevuparin (DF02) to treat patients with sickle-cell disease
  • Agios Pharmaceuticals, Inc. for its investigational medicine AG-348 for the treatment of pyruvate kinase deficiency
  • Treeway for its investigational treatment for amyotrophic lateral sclerosis
  • Aduro Biotech, Inc. for CRS-207, an immuno-oncology product candidate, for the treatment of mesothelioma

Recent Fast Track Designations

  • Insys Therapeutics for pharmaceutical cannabidiol for the treatment of Dravet syndrome
  • Axsome Therapeutics for AXS-02 drug to treat pain associated with complex regional pain syndrome
  • Heat Biologics for HS-410 in combination with Bacillus Calmette-Guérin immunotherapy for the treatment of non-muscle invasive bladder cancer

Recent Breakthrough Therapy Designations

  • Celldex Therapeutics for its brain cancer immunotherapy Rintega to treat EGFRvIII-positive glioblastoma
  • Memorial Sloan Kettering Cancer Center for Atara’s cytotoxic T lymphocytes activated against Epstein-Barr virus in the treatment of patients with rituximab-refractory, EBV-associated lymphoproliferative disease