Navigation Links
Enobia Pharma Receives FDA Orphan Drug Designation for Hypophosphatasia Product Candidate ENB-0040
Date:9/19/2008

MONTREAL, Sept. 19 /PRNewswire/ -- Enobia Pharma, an emerging biotech company focused on developing novel therapeutics for serious bone disorders, announced that it has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for ENB-0040, its enzyme replacement therapy (ERT) for hypophosphatasia, a rare, life-threatening genetic bone disease. In August 2008, Enobia dosed the first patient in a Phase I clinical trial of ENB-0040.

"ENB-0040 represents a potential drug therapy for the patients with hypophosphatasia, an under-recognized disease that can be fatal in infants and cause serious disability in older patients that has no currently approved FDA treatment," said Robert Heft PhD, Chief Executive Officer of Enobia. "The recent dosing of the first adult hypophosphatasia patient in our Phase I clinical trial and receipt of orphan drug designation are important milestones for this program."

The FDA grants Orphan Drug Designation to encourage biotechnology and pharmaceutical companies to develop products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 people in the United States. This designation will entitle Enobia to a seven-year period of marketing exclusivity for the drug upon FDA approval, as well as the opportunity to apply for funding from the U.S. government to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential waiver of the FDA's application user fee.

About Hypophosphatasia

Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Patients have low levels of the tissue non-specific form of alkaline phosphatase, an important regulator of bone mineralization, leading to rickets in infants and children and osteomalacia ("soft bones" resulting from poor mineralization) in adults. Disease severity is inversely proportional to the age at symptom onset, but morbidity can be cumulative and worsen with age. Clinical severity ranges from the severe perinatal or infantile form, with profound skeletal hypomineralization and respiratory compromise often causing death, to a more slowly progressive and debilitating osteomalacia in adults.

In the infantile form, infants may appear normal at birth but develop serious symptoms in the first six months of life. These can include failure to thrive, respiratory failure, fractures, and seizures. Radiographic findings include generalized hypomineralization and rickets. Mortality in these patients may be as high as 50%. In the childhood form, patients have varying degrees of hypomineralization, frank rickets, short stature, bone pain, muscle weakness, delayed motor milestones, early loss of deciduous teeth, and may experience frequent, poorly-healing fractures. In the adult form, the underlying osteomalacia causes bone pain due to overt or poorly-healing stress fractures that in some cases stops ambulation.

About ENB-0040

ENB-0040 is a fusion protein that includes the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP), an immunoglobulin Fc domain and a patented anionic peptide used to target the enzyme to bone. Preclinical studies of ENB-0040 in the "knockout" mouse model of severe hypophosphatasia were recently published in the Journal of Bone and Mineral Research [June 2008:23:777-787] and showed that subcutaneous administration of ENB-0040 significantly improved survival and prevented the skeletal and dental manifestations of the disease.

About Enobia Pharma Inc.

Enobia Pharma Inc., is a private, Montreal based company focused on the development of therapeutics to treat serious bone disorders for which there is no currently approved drug therapy. Enzyme Replacement Therapy for the treatment of hypophosphatasia is the Company's lead program. In 2007 Enobia completed a $40M Series B financing lead by OrbiMed Advisors and CTI Life Sciences.

Company Contact: Julie Anne Smith, (514) 596-2901, extension 214


'/>"/>
SOURCE Enobia Pharma
Copyright©2008 PR Newswire.
All rights reserved

Related medicine technology :

1. Enobia Pharma Initiates Clinical Testing of Enzyme Replacement Therapy to Treat Hypophosphatasia
2. Pharmasset to Present at the UBS Global Life Sciences Conference on Wednesday, September 24th
3. Memory Pharmaceuticals and Roche Expand R3487/MEM 3454 Development Program
4. Oramed Pharmaceuticals Launches GLP1-Analog Program, Based on its Proprietary Drug Delivery Platform
5. Cadence Pharmaceuticals Updates Omigard(TM) Clinical Timeline
6. VIA Pharmaceuticals to Present at American Heart Association Scientific Sessions 2008
7. Unibioscreen and Drais Pharmaceuticals Partner to Develop and Commercialize UNBS 5162 for Treatment of Cancer
8. Vanda Pharmaceuticals Provides Update on Iloperidone
9. Cerimon Pharmaceuticals Announces Positive Results from a Phase II Clinical Study of Its Diclofenac Sodium Patch for Acute Pain
10. Jazz Pharmaceuticals, Inc. Announces Final Patient Has Completed Phase III Clinical Trial of Sodium Oxybate to Treat Fibromyalgia
11. Addrenex Pharmaceuticals Announces Positive Phase III Clinical Results for Clonicel to Treat ADHD
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/24/2016)... , June 24, 2016   Pulmatrix, Inc ... company developing innovative inhaled drugs, announced today that it ... Russell Investments reconstituted its comprehensive set of U.S. ... "This is an important milestone for Pulmatrix," said ... increase shareholder awareness of our progress in developing drugs ...
(Date:6/23/2016)... , June 23, 2016  MedSource announced today ... its e-clinical software solution of choice.  This latest ... possible value to their clients by offering a ... preferred relationship establishes nowEDC as the EDC platform ... MedSource,s full-service clients.  "nowEDC has long been a ...
(Date:6/23/2016)... , June 23, 2016 Research ... "Pharmaceutical Excipients Market by Type (Organic Chemical (Sugar, Petrochemical, ... (Oral, Topical, Coating, Parenteral) - Global Forecast to 2021" ... The global pharmaceutical excipients market is ... a CAGR of 6.1% in the forecast period 2016 ...
Breaking Medicine Technology:
(Date:6/24/2016)... ... 24, 2016 , ... Those who have experienced traumatic events may suffer from ... avenues, such as drug or alcohol abuse, as a coping mechanism. To avoid this ... coping following a traumatic event. , Trauma sufferers tend to feel a range of ...
(Date:6/24/2016)... ... 24, 2016 , ... The Pulmonary Hypertension Association (PHA) learned ... receive two significant new grants to support its work to advance research and ... by recognizing patients, medical professionals and scientists for their work in fighting pulmonary ...
(Date:6/24/2016)... ... 24, 2016 , ... Comfort Keepers® of San Diego, CA is excited to ... Recovery® program to drive cancer patients to and from their cancer treatments. Comfort ... quality of life and ongoing independence. Getting to and from medical treatments is ...
(Date:6/24/2016)... ... June 24, 2016 , ... EB Medicine ... Making in Emergency Medicine conference in Ponte Vedra Beach, FL. The awards honor ... Emergency Medicine Practice and Pediatric Emergency Medicine Practice. , “With this ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... ... is actively feeding the Frederick area economy by obtaining investment capital for emerging ... the past 2½ years that have already resulted in more than a million ...
Breaking Medicine News(10 mins):