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Specialty Pharma Journal Specialty, Precision & Technology

Breaking News

FDA Approves Gavreto for the Treatment of Adults with Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer
Incyte Announces Pivotal GEOMETRY mono-1 Study Results of Capmatinib in Patients with METex14 Metastatic Non-Small Cell Lung Cancer Published in NEJM
Natera Announces SMART Study Data for Aneuploidy and 22q Unblinded
Aimmune Agrees to be Acquired by Nestlé Health Science for $2 Billion
Twist Bioscience Announces Potent SARS-CoV-2 Neutralizing Data from COVID-19 Therapeutic Antibody Program
Final Analysis of the Observational GioTag Study: Sequential Afatinib and Osimertinib in Patients with EGFR Mutation-Positive NSCLC
Santhera Exercises Option to Obtain Worldwide Rights to Vamorolone in Duchenne Muscular Dystrophy and All Other Indications
FDA Approves Onureg, a New Oral Therapy, as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia
FDA Accepts Vertex’s sNDA for Trikafta, Symdeko and Kalydeco for Additional CFTR Mutations
Gilead Sciences and Jounce Therapeutics Announce Exclusive License Agreement for Novel Immunotherapy Program

Tag Archives: Niemann-Pick Type C1 Disease

August, 2016

31 August
System May Help Treat Rare Genetic Disorder, Reduce Severe Side Effects

PORTLAND, Ore. – Researchers at Oregon State University and other institutions have discovered a type of drug delivery system that may offer new hope for patients with a rare, ultimately fatal genetic disorder – and make what might become a terrible choice a little easier. No treatment currently exists for …
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January, 2016

6 January
FDA Grants Breakthrough Therapy Designation to Vtesse’s Drug for Treatment of a Rare Genetic Disease

GAITHERSBURG, Md., Jan. 6, 2016 /PRNewswire/ — Vtesse, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted its drug candidate, VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC), Breakthrough Therapy designation status. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status …
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