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Rare Diseases

Data from Alnylam’s Phase 1 Trial of Givosiran Published in The New England Journal of Medicine

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that results from the Phase 1 study of givosiran, an investigational, subcutaneous RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), were published online todayin The New England Journal of Medicine (NEJM). The …

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Zogenix Submits Fintepla Applications to the FDA and EMA for the Treatment of Dravet Syndrome

EMERYVILLE, Calif., Feb. 06, 2019 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ:ZGNX), a global pharmaceutical company developing rare disease therapies, today announced it has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) and submitted a Marketing Authorization Application (MAA) to the …

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Amicus Therapeutics Announces Positive data in Pompe Disease Phase 1/2 Study for Up to 24 Months

CRANBURY, N.J., and ORLANDO, Fla., Feb. 05, 2019 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive results from a global Phase 1/2 clinical study (ATB200-02) to investigate AT-GAA in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen (disease substrate) …

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Apellis’ APL-2 Receives Orphan Drug Designation from the FDA for the Treatment of Autoimmune Hemolytic Anemia

CRESTWOOD, Ky., and WALTHAM, Mass., Feb. 05, 2019 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals Inc. (Nasdaq:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through inhibition of the complement system, today announced that its C3 complement inhibitor, APL-2, has been granted orphan drug designation by the U.S. Food …

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FDA Grants Priority Review to Daiichi Sankyo’s CSF1R Inhibitor for Treatment of Patients with Tenosynovial Giant Cell Tumor

TOKYO and MUNICH and BASKING RIDGE, N.J., Feb. 5, 2019 /PRNewswire/ — Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) and granted Priority Review for pexidartinib for the treatment of adult patients with symptomatic tenosynovial …

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REGENXBIO Receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy for Treatment of CLN2 Form of Batten Disease

ROCKVILLE, Md., Jan. 31, 2019 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced the U.S. Food and Drug Administration(FDA) granted Rare Pediatric Disease Designation to RGX-181. RGX-181 is a one-time treatment candidate for …

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FDA Grants Orphan Drug Designation to Prana’s PBT434 for Treatment of Multiple System Atrophy

MELBOURNE, Australia & SAN FRANCISCO–(BUSINESS WIRE)–Prana Biotechnology Ltd (ASX: PBT, NASDAQ: PRAN) (“Prana” or “the Company”) has today announced the US Food and Drug Administration (FDA) has granted Orphan Drug designation for its lead molecule, PBT434, for the treatment of Multiple System Atrophy (MSA). This is the first time the …

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Aldeyra Therapeutics Expands Retinal Disease Pipeline With Acquisition Of Helio Vision

LEXINGTON, Mass., Jan. 29, 2019 /PRNewswire/ — Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to developing and commercializing next-generation medicines to improve the lives of patients with immune-mediated diseases, today announced that it has acquired Helio Vision, Inc. (Helio Vision), a privately held biotechnology company. The acquisition adds to Aldeyra’s pipeline a Phase 3-ready …

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Alexion Announces Positive Top-Line Results from Phase 3 Study Evaluating Ultomiris in Complement Inhibitor-Naïve Patients with atypical Hemolytic Uremic Syndrome

BOSTON–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Phase 3 study of ULTOMIRIS™ (ravulizumab-cwvz), the company’s long-acting C5 complement inhibitor, met its primary objective in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome (aHUS). In the initial 26 week treatment period, 53.6 percent of patients (95% CI [39.6%, 67.5%]) demonstrated …

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Alnylam and CAMP4 Therapeutics Enter Rare Disease Research Collaboration

CAMBRIDGE, Mass.–(BUSINESS WIRE)–CAMP4 Therapeutics today announced a research collaboration with Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) focused on identifying new druggable targets to address an undisclosed rare disease of the liver with a significant unmet need. The collaboration brings together CAMP4’s expertise in rapidly pinpointing signaling targets that control the expression …

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