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:10/12/2017)... Oct. 12, 2017 West Pharmaceutical Services, ... solutions for injectable drug administration, today announced that it ... opens on Thursday, October 26, 2017, and will follow ... business expectations at 9:00 a.m. Eastern Time. To participate ... (International). The conference ID is 94093362. ...
(Date:10/11/2017)... Oct. 11, 2017  Caris Life Sciences ® , ... the promise of precision medicine, today announced that St. ... Precision Oncology Alliance™ (POA) as its 17 th ... St. Jude Crosson Cancer Institute will help develop standards ... of tumor profiling, making cancer treatment more precise and ...
(Date:10/11/2017)... , Oct. 11, 2017  Hill-Rom Holdings, Inc. ... Aspen Surgical facility in Las Piedras, Puerto ... scalpels and blades. ... that the facility sustained minor structural damage, temporary loss ... Maria. Repairs have been completed, manufacturing operations have resumed, ...
Breaking Medicine Technology:
(Date:10/13/2017)... ... October 13, 2017 , ... Many families have long-term insurance that covers ... companies have a waiver for care if the client has a cognitive impairment diagnosis. ... pays for care, is often waived, so the benefits from their insurance start immediately,” ...
(Date:10/13/2017)... ... October 13, 2017 , ... Talented host, ... lowdown on sciatica in a new episode of "Success Files," which is an ... and innovation and investigates each subject in-depth with passion and integrity. , Sciatica ...
(Date:10/12/2017)... ... October 12, 2017 , ... CitiDent and San Francisco ... using cutting-edge Oventus O2Vent technology. As many as 18 million Americans are estimated ... in breathing. Oral appliances can offer significant relief to about 75 percent of ...
(Date:10/12/2017)... ... ... Asante, a nationally recognized health system in southern Oregon, ... health joint venture through an agreement, effective October 1, 2017, to create AccentCare ... company with Asante, delivering clinically integrated care, for the past eight years. This ...
(Date:10/12/2017)... ... October 12, 2017 , ... On Saturday, October 21, the Health & ... by Moonlight to raise money for the American Heart Association Heart Walk. Teams of ... will work together to keep their treadmills moving for 5 hours. Treadmills will start ...
Breaking Medicine News(10 mins):