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Latest Orphan drug Stories

2014-08-06 16:28:42

Input will help shape the role of public contributions to orphan drug approvals OTTAWA, Aug. 6, 2014 /CNW/ - The Honourable Rona Ambrose, Minister of Health, announced today that the Government of Canada has launched a pilot project targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs. As announced in October of 2012, Health Canada has been developing an Orphan Drug Framework to spur innovation and research into new treatments...

2014-08-05 08:33:19

SAN FRANCISCO, Aug. 5, 2014 /PRNewswire/ -- Perlstein Lab PBC, a San Francisco-based public benefit corporation whose mission is precision orphan drug discovery, just closed a $2 million seed round. The round was co-led by the clinical-stage orphan drug developer Retrophin, Inc. and the Wilsey family, which is personally affected by an orphan disease. http://photos.prnewswire.com/prnvar/20140805/133384 One in 12 people suffer from an orphan disease, yet 95% of orphan diseases have...

2014-08-05 04:21:27

NESS ZIONA, Israel, August 5, 2014 /PRNewswire/ -- Atox Bio today announced that the European Commission has granted AB103 (Sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH) orphan medicinal product designation (EU/3/14/1294) for the treatment of necrotizing soft tissue infections. Atox Bio is represented in the EU by Dr. Ulrich Granzer as agent. In addition to a 10-year period of marketing exclusivity in the EU after...

2014-08-05 00:21:03

SAN DIEGO, Aug. 5, 2014 /PRNewswire/ -- Genoa Pharmaceuticals, the leader in inhaled medicines for pulmonary fibrosis, today announced the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to Genoa for the use of pirfenidone in their lead program - inhaled GP-101 for the treatment of IPF. "Acquiring orphan status marks an important regulatory milestone in GP-101's life cycle to treat people with this devastating disease," said Mark Surber, Ph.D., Genoa's...

2014-08-04 08:28:32

Significant company milestones include completion of the Phase 2 PUNCH(TM) CD clinical trial and FDA designation of RBX2660 as Orphan Drug and Fast Track ROSEVILLE, Minn., Aug 4, 2014 /PRNewswire/ -- Rebiotix Inc. announced this morning that it has raised a total of $25 million in Series B Funding. The total funds raised since inception by the clinical-stage biotechnology company founded in 2011 is $30 million. Rebiotix will use the additional funds to support pivotal clinical...

2014-07-29 08:32:30

BELLEVILLE, ON, July 29, 2014 /PRNewswire/ - Bioniche Life Sciences Inc. (TSX: BNC) today announced the submission to the US Food and Drug Administration (FDA) Office of Orphan Products Development, an application for Orphan Drug Designation for its Mycobacterial Cell Wall-Nucleic Acid Complex (MCNA) sterile suspension for the treatment of patients with Bacillus Calmette-Guérin (BCG) refractory/relapsing high grade non-muscle invasive bladder cancer. The decision to proceed...

2014-07-22 08:32:03

- Neurotrope enters into world-wide, exclusive license agreement with Mount Sinai; Files related provisional patent for Bryostatin-1 - PLANTATION, Fla., July 22, 2014 /PRNewswire/ -- Neurotrope Bioscience, Inc., a subsidiary of Neurotrope, Inc. (OTCQB: NTRP) has signed a world-wide, exclusive license agreement with the Icahn School of Medicine at Mount Sinai (Mount Sinai) to utilize its proprietary information and data package for the use of Bryostatin-1 in the treatment of...

2014-07-07 08:27:35

ROCKVILLE, Md., July 7, 2014 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a clinical-stage pharmaceutical company developing therapeutics for the treatment of cancer and other diseases, announced today that its orally-active, Aurora A/angiogenic kinase inhibitor, ENMD-2076, has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma (HCC). http://photos.prnewswire.com/prnvar/20140706/124566...

2014-07-02 12:26:27

LONDON, July 2, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report: Global Orphan Drugs Market 2014-2018http://www.reportbuyer.com/pharma_healthcare/therapeutic/global_orphan_drugs_market_2014_2018.html TechNavio's analysts forecast the Global Orphan Drugs market to grow at a CAGR of 5.67 percent over the period 2013-2018. One of the key factors contributing to this market growth is the shorter development timelines. The Global Orphan Drugs market has also...

2014-06-24 12:27:50

ST. PETERSBURG, Fla., June 24, 2014 /PRNewswire-USNewswire/ -- InformedDNA announced today a strategic new collaboration with Edimer Pharmaceuticals to address a critical research need for patients with X-linked Hypohidrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder. The partnership will allow InformedDNA to identify patients with XLHED and support Edimer's clinical programs. Under the agreement, InformedDNA has developed a genetic counseling program for patients and...


Word of the Day
endocarp
  • The hard inner (usually woody) layer of the pericarp of some fruits (as peaches or plums or cherries or olives) that contains the seed.
This word comes from the Greek 'endon,' in, within, plus the Greek 'kardia,' heart.
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