Specialty Pharma Journal Specialty, Precision & Technology
Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will …
Tag Archives: Breakthrough Therapy
July, 2018
May, 2018
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24 May
Pfizer’s Tafamidis Receives Breakthrough Therapy Designation from the FDA for Treatment of Transthyretin Cardiomyopathy
NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) announced today that tafamidis received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for the treatment of patients with transthyretin cardiomyopathy, a rare, fatal, and underdiagnosed condition associated with progressive heart failure.1,2 This decision is supported by topline results from the tafamidis …
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24 May
FDA Grants Breakthrough Therapy Designation to Lenti-D for the Treatment of Cerebral Adrenoleukodystrophy
CAMBRIDGE, Mass.–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Lenti-D™ for the treatment of patients with cerebral adrenoleukodystrophy (CALD), a rare, serious and life-threatening hereditary neurological disorder. Breakthrough Therapy designation is designed to expedite the development and …
January, 2018
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5 January
FDA Grants Breakthrough Therapy Designation to Novartis’ Promacta for Treatment of Severe Aplastic Anemia
Basel, January 4, 2018 – Novartis today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Promacta®(eltrombopag) for use in combination with standard immunosuppressive therapy for the treatment of patients with severe aplastic anemia (SAA) as a first-line therapy. Promacta, which is marketed as Revolade® in most …
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3 January
FDA Grants Breakthrough Therapy Designation to Novartis’ Breast Cancer Drug Kisqali
Basel, January 3, 2018 – Novartis today announced Kisqali® (ribociclib) received US Food and Drug Administration (FDA) Breakthrough Therapy designation for initial endocrine-based treatment of pre- or perimenopausal women with hormone-receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic breast cancer in combination with tamoxifen or an aromatase inhibitor. This …
December, 2017
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21 December
FDA Grants Breakthrough Therapy Designation for Avelumab in Combination with Inlyta in Advanced Renal Cell Carcinoma
DARMSTADT, Germany and NEW YORK, December 21, 2017 /PRNewswire/ — Second Breakthrough Therapy Designation for avelumab in hard-to-treat cancer Renal cell carcinoma, the most common form of kidney cancer, has a poor prognosis in advanced stage[1],[2] Javelin Renal clinical development program is ongoing, including Phase III first-line study Merck and …
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18 December
FDA Grants Breakthrough Therapy Designation to Novartis’ Fingolimod for Pediatric Multiple Sclerosis
EAST HANOVER, N.J., Dec. 18, 2017 /PRNewswire/ — Novartis today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for fingolimod for the treatment of children and adolescents 10 years of age or older with relapsing multiple sclerosis (MS). Fingolimod, also known as Gilenya® in …
September, 2017
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8 September
Regeneron and Sanofi’s Cemiplimab Receives FDA Breakthrough Therapy Designation for Cutaneous Squamous Cell Carcinoma
TARRYTOWN, N.Y. and PARIS, Sept. 8, 2017 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to cemiplimab (REGN2810) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC, the second deadliest skin cancer …
August, 2017
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7 August
FDA Grants Priority Review and Breakthrough Therapy Designation for Zelboraf in Erdheim-Chester Disease with BRAF V600 Mutation
SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Zelboraf® (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD …
July, 2017
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31 July
FDA Grants Breakthrough Therapy Designation to Imfinzi for Non-Small Cell Lung Cancer
AstraZeneca and MedImmune, its global biologics research and development arm, today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Imfinzi (durvalumab) for the treatment of patients with locally-advanced, unresectable non-small cell lung cancer (NSCLC) whose disease has not progressed following platinum-based chemoradiation therapy. Sean Bohen, …