Specialty Pharma Journal Specialty, Precision & Technology
Cambridge, Mass. – November 13, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG) the global leader in rare diseases, announced today the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental new drug application to extend the indication of GATTEX® (teduglutide [rDNA origin]) for Injection to pediatric patients (aged 1-17 years …
Tag Archives: rare diseases
November, 2018
October, 2018
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26 October
Ultragenyx Announces Negative Topline Results from Phase 3 Study of UX007 in Patients with Glut1 DS with Disabling Movement Disorders
NOVATO, Calif., Oct. 26, 2018 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced its Phase 3 study of UX007 in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS) experiencing disabling …
September, 2018
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21 September
Amicus Acquires Gene Therapy Portfolio of Ten AAV Programs in Neurologic Lysosomal Storage Disorders
CRANBURY, N.J., Sept. 20, 2018 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced the signing of a definitive agreement in which Amicus Therapeuticswill receive worldwide development and commercial rights for ten gene therapy programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. The ten programs are licensed …
August, 2018
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24 August
PTC Therapeutics Successfully Completes Acquisition of Agilis Biotherapeutics
SOUTH PLAINFIELD, N.J., Aug. 23, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ : PTCT ), today announced that it has successfully completed the acquisition of Agilis Biotherapeutics, Inc., a private biotechnology company focused on the advancement of innovative gene therapy programs for rare genetic disorders that affect the central nervous …
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23 August
FDA Approves Dompé’s Oxervate for Treatment of Neurotrophic Keratitis, a Rare Eye Disease
MILAN and SAN BRUNO, Calif., Aug. 22, 2018 /PRNewswire/ — Dompé today announced that the U.S. Food and Drug Administration (FDA) has approved OxervateTM (cenegermin-bkbj ophthalmic solution), a breakthrough therapy for neurotrophic keratitis (NK), a rare and progressive eye disease that can lead to corneal scarring and vision loss.1 “Neurotrophic …
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6 August
Mallinckrodt Forms Collaborative Research Partnership with Washington University Focused on Rare Diseases
STAINES-UPON-THAMES, United Kingdom, Aug. 6, 2018 /PRNewswire/ — Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced it has formed a collaborative research partnership with Washington University School of Medicine in St. Louis, a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News …
July, 2018
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5 July
Enzyvant and Visikol Collaborate to Develop Innovative Digital Pathology Assay to Support RVT-802 Development
BASEL, Switzerland and WHITEHOUSE STATION, N.J., July 5, 2018 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced that it has formed a partnership with Visikol, a contract research organization focused on digital pathology and drug discovery. Under the terms of …
May, 2018
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25 May
FDA Approves Biomarin’s Palynziq Injection for Treatment of Phenylketonuria, A Rare Genetic Disease
SAN RAFAEL, Calif., May 24, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that BioMarin received standard approval from the U.S. Food and Drug Administration (FDA) for Palynziq™ (pegvaliase-pqpz) Injection to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 micromol/L …
April, 2018
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18 April
FDA Approves Crysvita, The First Therapy for X-Linked Hypophosphatemia, A Rare Inherited Form of Rickets
NOVATO, Calif. and TOKYO and LONDON, April 17, 2018 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co. Ltd(Kyowa Hakko Kirin), and Kyowa Kirin International PLC (Kyowa Kirin International) today announced that the U.S. Food and Drug Administration (FDA) has approved Crysvita® (burosumab-twza) for …
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13 April
GSK Transfers Its Portfolio of Rare Disease Gene Therapies to Orchard Therapeutics
GSK and Orchard Therapeutics today announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for patients. This acquisition strengthens Orchard’s position as a global leader in gene therapy for …