Orphan Drugs
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Orphan Drugs
pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease
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Orphan Drug Designation for the use of Teicoplanin in Cystic Fibrosis — CheckOrphan

Orphan Drug Designation for the use of Teicoplanin in Cystic Fibrosis — CheckOrphan | Orphan Drugs | Scoop.it
Orphan Drug Designation for the use of Teicoplanin in Cystic Fibrosis
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AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1 - Financial Buzz

AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1 - Financial Buzz | Orphan Drugs | Scoop.it
SAKIGAKE is intended to promote research and development in Japan for innovative new medical products that satisfy certain criteria, such as the severity of the intended indication, by providing prioritized consultation review during the early stages of development and by shortening the target review period for license applications from 12 months to as few as six months. The benefits of SAKIGAKE Designation are similar to the Breakthrough Therapy Designation in the United States and access into the PRIority MEdicines (PRIME) scheme in the EU, both of which have already been granted to AVXS-101.
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Orphan drug status for Astellas Pharma’s gilteritinib in Japan for the treatment of FLT3mut+ acute myeloid leukemia (AML)

Orphan drug status for Astellas Pharma’s gilteritinib in Japan for the treatment of FLT3mut+ acute myeloid leukemia (AML) | Orphan Drugs | Scoop.it
Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Drug designation to gilteritinib for the treatment of FLT3mut+ acute myeloid leukemia (AML), the drug’s prospective indication. The positive news for the drug’s developer, Astellas Pharma (TYO: 4503), saw … Continued
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Elysium Health™ Granted Orphan Drug Designation for EH301 to Treat Amyotrophic Lateral Sclerosis

Elysium Health™ Granted Orphan Drug Designation for EH301 to Treat Amyotrophic Lateral Sclerosis | Orphan Drugs | Scoop.it
NEW YORK, March 29, 2018 /PRNewswire/ -- Elysium Health™ Granted Orphan Drug Designation for EH301 to Treat Amyotrophic Lateral Sclerosis (ALS).
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Martin Pharmaceuticals Receives FDA Orphan Drug Designation For Livantra

Martin Pharmaceuticals Receives FDA Orphan Drug Designation For Livantra | Orphan Drugs | Scoop.it
NewsFDA grants Orphan Drug Designation for Livantra in the treatment of acute on chronic liver failure.Contributed Author: Martin PharmaceuticalsTopics: Drug Development
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Cynata’s GvHD treatment receives Orphan Drug Designation

Cynata’s GvHD treatment receives Orphan Drug Designation | Orphan Drugs | Scoop.it
Regenerative medicine company Cynata Therapeutics has announced that the US Food and Drug Administration (FDA) has given Orphan Drug Designation...Rea
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announces orphan drug designation for ARGX-113 for the treatment of myasthenia gravis in Europe - Argenx

announces orphan drug designation for ARGX-113 for the treatment of myasthenia gravis in Europe - Argenx | Orphan Drugs | Scoop.it
Argenx discovers, designs and develops innovative antibody therapeutics for its own pipeline of treatments for cancer and autoimmune diseases and for its partners. Harnessing our technology and know-how, we aim to meet the needs of patients by engineering antibodies to target diseases too complex for other antibody technologies.
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Fda grants pluristem orphan drug designation for its plx-r18 cell therapy as treatment for acute radiation syndrome - news press release pharmiweb.com

CITY, Yisrael, October. 19, 2017 (FIELD NEWSWIRE) -- Pluristem Therapeutics Opposition. (NASDAQ: PSTI) (TASE: PSTI), a salient develope
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Amicus Therapeutics Announces Approval of Galafold® (Migalastat) for Fabry Disease in Japan - News Press Release | PharmiWeb.com

Amicus Therapeutics Announces Approval of Galafold® (Migalastat) for Fabry Disease in Japan - News Press Release | PharmiWeb.com | Orphan Drugs | Scoop.it

CRANBURY, N.J., March 22, 2018 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) announced today that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved the oral small molecule pharmacological chaperone Galafold® capsules 123m
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Soligenix Receives Orphan Drug Designation from the European Commission for RiVax® for Prevention of Ricin Poisoning - Financial Buzz

Soligenix Receives Orphan Drug Designation from the European Commission for RiVax® for Prevention of Ricin Poisoning - Financial Buzz | Orphan Drugs | Scoop.it
<span class="xn-location">PRINCETON, N.J.</span>, <span class="xn-chron">March 22, 2018</span> /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the European Commission, acting on the positive recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products, has granted orphan drug designation to the Company's recombinant modified ricin toxin A-chain subunit (the active pharmaceutical ingredient in RiVax<sup>®</sup>) for the prevention of ricin poisoning.  RiVax<sup>®</sup> has previously been granted orphan drug designation from the US Food and Drug Administration (FDA).
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Cellectar Biosciences Granted Orphan Drug Designation for CLR 131 to Treat Neuroblastoma Nasdaq:CLRB

Cellectar Biosciences Granted Orphan Drug Designation for CLR 131 to Treat Neuroblastoma Nasdaq:CLRB | Orphan Drugs | Scoop.it
Media Files Cellectar Biosciences, Inc.Logo LOGO URL | Copy the link below Formats available: Original Medium Small...
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Tetra Bio-Pharma Gets FDA Orphan Drug Designation for PPP001

Tetra Bio-Pharma Gets FDA Orphan Drug Designation for PPP001 | Orphan Drugs | Scoop.it
Tetra Bio-Pharma announced that the FDA granted an orphan drug designation to the active ingredients of its PPP001 drug treating complex regional pain syndrome.
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Exelixis’ Partner Ipsen Announces EMA Validation of the Application for a New Indication for CABOMETYX® (cabozantinib) for Previously Treated Advanced Hepatocellular Carcinoma

Exelixis’ Partner Ipsen Announces EMA Validation of the Application for a New Indication for CABOMETYX® (cabozantinib) for Previously Treated Advanced Hepatocellular Carcinoma | Orphan Drugs | Scoop.it
Wednesday, March 28th 2018 at 5:05am UTC – In the pivotal phase 3 CELESTIAL trial, CABOMETYX provide
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GeneTx Biotherapeutics receives FDA orphan-drug designation for Angelman syndrome treatment

GeneTx Biotherapeutics receives FDA orphan-drug designation for Angelman syndrome treatment | Orphan Drugs | Scoop.it
DOWNERS GROVE, Ill., March 27, 2018 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration has granted orphan-drug designation to GeneTx Biotherapeutics LLC’s GTX-101 fo
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Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy

Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy | Orphan Drugs | Scoop.it
Thursday, March 29th 2018 at 11:00am UTC CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar Rock, a biophar
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Czech-made medicine gains EMA's orphan drug status | Prague Monitor

Czech-made medicine gains EMA's orphan drug status | Prague Monitor | Orphan Drugs | Scoop.it
ČTK | 29 MARCH 2018 Prague, March 28 (CTK) - A medicine developed by the firm Sotio, a part of the PPF Group, and designated for the treatment of ovarian cancer, is the first Czech-made medicine to have gained the orphan drug status from the European Medicines Agency (EMA), Sotio said in a press...
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GeneTx Biotherapeutics receives FDA orphan-drug designation for Angelman syndrome treatment - Financial Buzz

GeneTx Biotherapeutics receives FDA orphan-drug designation for Angelman syndrome treatment - Financial Buzz | Orphan Drugs | Scoop.it
DOWNERS GROVE, Ill., March 27, 2018 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration has granted orphan-drug designation to GeneTx Biotherapeutics LLC’s GTX-101 for the treatment of Angelman syndrome, a rare neurogenetic disorder that affects approximately one in 15,000 people. GTX-101 is the first drug candidate for the startup biotechnology company.<br />
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MaxiNovel Pharmaceuticals, Inc. Announces FDA Orphan Drug Designation for MAX-40279 for the Treatment of Acute Myeloid Leukemia (AML) | おいしい美食生活

MaxiNovel Pharmaceuticals, Inc. Announces FDA Orphan Drug Designation for MAX-40279 for the Treatment of Acute Myeloid Leukemia (AML) | おいしい美食生活 | Orphan Drugs | Scoop.it
GUANGZHOU, China--(BUSINESS WIRE)--MaxiNovel Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration ("FDA") has granted MaxiNovel Orphan Drug Designation for MAX-40279 in the treatment of Acute Myeloid Leukemi
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Krystal to File IND Application for Dystrophic EB Investigational Gene Therapy KB103

Krystal to File IND Application for Dystrophic EB Investigational Gene Therapy KB103 | Orphan Drugs | Scoop.it
Read more about Krystal Biotech’s lead gene therapy KB103 and the advances made to initiate Phase 1/2 clinical trials as a treatment for dystrophic epidermolysis bullosa.
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BIOPHYTIS files for Orphan Drug Designation for Sarconeos in Duchenne Muscular Dystrophy (DMD) Paris Stock Exchange:ALBPS

BIOPHYTIS files for Orphan Drug Designation for Sarconeos in Duchenne Muscular Dystrophy (DMD) Paris Stock Exchange:ALBPS | Orphan Drugs | Scoop.it
Media Files BIOPHYTIS SA Logo LOGO URL | Copy the link below Formats available: Original Medium Small...
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The FDA Gave Orphan Drug Designation to an Investigational Treatment For Cystic Fibrosis

The FDA Gave Orphan Drug Designation to an Investigational Treatment For Cystic Fibrosis | Orphan Drugs | Scoop.it
Read this story about a drug candidate for cystic fibrosis that recently received Orphan Drug Designation from the FDA.
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Trillium Therapeutics’ TTI-621 Receives Orphan Drug Designation for the Treatment of Cutaneous T-Cell Lymphoma Nasdaq:TRIL

Trillium Therapeutics’ TTI-621 Receives Orphan Drug Designation for the Treatment of Cutaneous T-Cell Lymphoma Nasdaq:TRIL | Orphan Drugs | Scoop.it
Media Files Trillium Therapeutics Inc.Logo LOGO URL | Copy the link below Formats available: Original Medium Small...
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TiGenix : and Takeda announce Alofisel® (darvadstrocel) receives approval to treat complex perianal fistulas in Crohn's disease in Europe | 4-Traders

TiGenix : and Takeda announce Alofisel® (darvadstrocel) receives approval to treat complex perianal fistulas in Crohn's disease in Europe | 4-Traders | Orphan Drugs | Scoop.it
PRESS RELEASE REGULATED INFORMATION INSIDE INFORMATION TiGenix and Takeda announce Alofisel® receives approval to treat complex perianal fistulas in Crohn's disease in Europe... | mars 23, 2018
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Revance announces orphan drug designation of rt002 for the treatment of cervical dystonia - thestreet

Revance Therapeutics, Opposition. (NASDAQ:RVNC), a bioengineering corporation antiemetic for child nonindustrial eubacteria toxin outpu
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Abeona Therapeutics Receives FDA Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease

Abeona Therapeutics Receives FDA Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease | Orphan Drugs | Scoop.it
The FDA has granted Rare Pediatric Disease Designation for the ABO-202 program (AAV-CLN1), an AAV-based gene therapy for the treatment of CLN1 disease (infantile and late infantile onset Batten disease).
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