Navigation Links
Hyperion Therapeutics Receives Orphan Drug Designation for HPN-100 for the Treatment of Urea Cycle Disorders

- Company preparing to initiate phase III clinical trial -

SOUTH SAN FRANCISCO, Calif., May 5 /PRNewswire/ -- Hyperion Therapeutics, Inc. today announced that HPN-100 has received orphan product designation from the U.S. Food and Drug Administration for maintenance treatment of patients with enzymes of the urea cycle. The Company recently announced results from its phase II study and intends to initiate a phase III clinical program in this indication in the second half of 2009.


HPN-100 is a pre-pro-drug of phenylacetic acid which is the active moiety of BUPHENYL(R), the only therapy currently FDA-approved as adjunctive therapy for the chronic management of patients with the most prevalent urea cycle deficiencies including those related to carbamylphosphate synthetase, ornithine transcarbamylase and argininosuccinic acid synthetase. HPN-100 is dosed orally in liquid form.

"Receiving orphan drug designation for HPN-100 in the treatment of urea cycle disorders is an important milestone and provides meaningful benefits to the Company as we work to complete clinical trials for this investigational compound," commented Donald Santel, Chief Executive Officer of Hyperion Therapeutics.

"The designation of HPN-100 as an orphan drug for the treatment of urea cycle disorders recognizes the unmet need that remains in this patient population," said Cindy LeMons, Executive Director of the National Urea Cycle Disorders Foundation (NUCDF). "We are encouraged by this designation and look forward to initiation of the phase III clinical trials."

About Orphan Drug Designation

Orphan drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance, waiver of Prescription Drug User Fee Act (PDUFA) filing fees, and a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication.

About Urea Cycle Disorders

Urea cycle disorders are inherited, inborn errors of metabolism present in an estimated 1 in 10,000 births in the United States. Patients with urea cycle disorders are deficient in one of the key enzymes that comprise the urea cycle, the body's primary vehicle for removing ammonia, a potent neurotoxin, from the bloodstream. Onset may occur at any age depending on the severity of the disorder. Left untreated, urea cycle disorders can cause dangerously heightened levels of ammonia in the bloodstream (hyperammonemia) resulting in brain damage, coma, and/or death.


The National Urea Cycle Disorders Foundation is a nationally-recognized nonprofit organization dedicated to saving children and adults from the catastrophic effects of urea cycle disorders. The Foundation is a lifeline to families and medical professionals all over the world desperately seeking crisis intervention and treatment information. The Foundation's mission is to provide information, guidance and support to patients and their families, to educate healthcare professionals on the identification, diagnosis and treatment of UCDs, to raise awareness so that no child or adult ever goes undiagnosed, and to stimulate and support critical research activities and a CURE. The Foundation is headquartered in Pasadena, California. For more information, visit, or call (626) 578-0833.


BUPHENYL is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). BUPHENYL should not be administered to patients with known hypersensitivity to sodium phenylbutyrate or any component of this preparation. The most common adverse reactions associated with BUPHENYL were amenorrhea dysfunction, decreased appetite, body odor (probably caused by its metabolite phenylacetate) and bad taste or taste aversion. Patients with urea cycle disorders should not take valproic acid, haloperidol, or steroids as these drugs have been reported to increase blood ammonia levels, and probenecid may affect the kidneys' excretion. Use with great care, if at all, in patients with congestive heart failure or severe renal insufficiency, and in clinical states where there is sodium retention with edema. Use caution when administering to patients with hepatic or renal insufficiency or inborn errors of beta oxidation. The safety or efficacy of doses in excess of 20 grams (40 tablets) per day has not been established.

About Hyperion Therapeutics

Hyperion Therapeutics is a privately held specialty therapeutics company focused on the development of therapies that address critical unmet needs and underserved patient populations in the areas of gastroenterology and hepatology. Hyperion and Ucyclyd Pharma, Inc., a subsidiary of Medicis Pharmaceutical Corporation, entered into a collaboration agreement for HPN-100 in August 2007. Under the terms of the agreement, Hyperion is conducting ongoing research and development of HPN-100 for urea cycle disorders, hepatic encephalopathy, and other forms of hyperammonemia. Hyperion is headquartered in South San Francisco, CA. For additional information, visit

BUPHENYL is a registered trademark of Ucyclyd Pharma, Inc.

Full Prescribing Information for BUPHENYL is available at or by contacting Ucyclyd Pharma, Inc.

SOURCE Hyperion Therapeutics, Inc.
Copyright©2009 PR Newswire.
All rights reserved

Related medicine technology :

1. Hyperion Therapeutics Announces Results for Phase II Study in Urea Cycle Disorders
2. Hyperion Therapeutics Completes Enrollment in Phase 1/2 Clinical Trial in Patients With Urea Cycle Disorders
3. William R. Ringo Joins Hyperion Therapeutics, Inc. Board of Directors as Chairman
4. Hyperion Therapeutics Announces Enrollment of First Patient in Phase 1/2 Clinical Trial of GT4P in Patients With Urea Cycle Disorders
5. Sirion Therapeutics Announces Clinical Data Presentations at the 2009 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting
6. Intarcia Therapeutics, Inc. Announces Completion of Enrollment of ITCA 650 Phase 1b Study for the Treatment of Type 2 Diabetes
7. Amicus Therapeutics Announces Issuance of New Plicera(TM) Composition of Matter Patent
8. Vantia Therapeutics Pipeline Continues to Mature
9. Calixa Therapeutics Announces Positive Phase 1 Results for CXA-101, a Novel Intravenous Cephalosporin Antibiotic with Excellent Anti-Pseudomonal Activity
10. Oxygen Biotherapeutics, Inc. Receives Letter From FDA Outlining Path Forward to Resume Oxycyte Clinical Trials in TBI in U.S.A.
11. Alba Therapeutics Corporation Announces the Next Clinical Trial of Its Lead Compound, Larazotide Acetate and Promotions
Post Your Comments:
(Date:11/24/2015)... Nov. 24, 2015  Ascendant Solutions, Inc. (Pink Sheets: ... Board of Directors has declared a special 1 percent stock ... is payable December 14, 2015, to shareholders of record December ... of additional shares of common stock. ... is a strong endorsement of our confidence in Ascendant,s growth ...
(Date:11/24/2015)... , Nov. 24, 2015 Diplomat Pharmacy, Inc. ... Vice President of Clinical Services, Education and Human Resources will ... webinar, "Oral Oncology Drugs: Health Plan Strategies for a Dynamic ... Beckie Fenrick , a consultant with the Cambridge Advisory ... The webinar will discuss the ...
(Date:11/24/2015)... , November 24, 2015 st  Scientific ... North America (RSNA) taking place in Chicago ... Booth 1122, Hall A. --> st  Scientific Assembly and ... (RSNA) taking place in Chicago ... Hall A. --> Molecular Dynamics will present its revolutionary ...
Breaking Medicine Technology:
(Date:11/24/2015)... Chicago, IL (PRWEB) , ... November 24, 2015 ... ... list are national leaders when it comes to several aspects of orthopedic care. ... as joint replacements, orthopedic surgeries and general orthopedic care. , Becker's Hospital ...
(Date:11/24/2015)... ... 2015 , ... Brillouin Energy Corp. Congress and Government officials ... renewable energy technologies capable of producing commercially useful amounts of thermal energy (heat) ... and HHT™ Boiler System reactor core modules were presented to Congress on Capitol ...
(Date:11/24/2015)... VA (PRWEB) , ... November 24, 2015 , ... ... people across the United States to support their local poison centers through donations ... designated as #GivingTuesday: calls it “a day that inspires people to collaborate ...
(Date:11/24/2015)... ... November 24, 2015 , ... Aided by seed funding from the Ron ... designed to yield insights into how to detect and treat pancreatic cancer (PC). ... from small, non-coding RNA molecules (ncRNA), genetic material that is present in the blood ...
(Date:11/24/2015)... ... , ... Dr. Rodney E. Willey , has answered a new calling – to relieve ... provides treatment for snoring and sleep apnea through oral appliance therapy. He ... Disorders in the US, one of four in the Illinois area. , Dr. Willey’s ...
Breaking Medicine News(10 mins):