FDA Designates Orphan Drug Status for Rare Diseases


The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. At the time of the enactment of the Orphan Drug Act, the United States Congress made several factual findings.  For example, the Congress observed that there are many diseases and conditions, such as Huntington’s disease, ALS (Lou Gehrig’s disease), Tourette syndrome, and muscular dystrophy, for example, which affect such small numbers of individuals that the diseases and conditions are considered rare in the United States.  As a result, adequate drugs have not been developed and realistically cannot be expected to be developed because companies would likely incur a financial loss in the pursuit of such drugs, given the high cost of developing drugs.

Back in the early 1980s when the Orphan Drug Act passed, Congress made the determination that a seven (7) year period of exclusivity was appropriate if a company produced a drug to treat a rare disease that affects less than 200,000 people in the United States.  Orphan designation is also possible if a disease affects more than 200,000 people if it can also be shown that there is no reasonable expectation that the cost of developing the drug could be recouped absent this period of exclusivity. This “patent-like” exclusivity is available even if the drug is not novel and non-obvious.

In addition to providing a seven-year term of market exclusivity upon final FDA approval, orphan drug designation allows the company receiving the designation to leverage a wide range of financial and regulatory benefits, including government and institutional grants, and waiver of FDA user fees for the potential submission of a New Drug Application.

Over the last several months, the FDA has granted Orphan designation to a number of interesting treatments, including the following.

1. Cystic Fibrosis Treatment

Novoteris, LLC., a clinical stage medical device and pharmaceutical developer, announced on October 9, 2013, that the Office of Orphan Products Development of the US Food and Drug Administration granted it orphan drug designation for the use of inhaled nitric oxide for the treatment of Cystic Fibrosis. Cystic fibrosis is an inherited disease characterized by the buildup of thick, sticky mucus that can damage many of the body’s organs. The disorder’s most common signs and symptoms include progressive damage to the respiratory system and chronic digestive system problems.

Chris Miller, PhD, Chief Science Advisor, noted that, “The results from our pilot trial demonstrated more than a 15% increase in lung function following two weeks of therapy and we anticipate that we will be able to demonstrate similar outcomes in our Phase II trials.”

2. Management of Hepatocellular Carcinoma

Guerbet LLC, USA announced on October 21, 2013, that it has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for Lipiodol (Ethiodized Oil) Injection for management of patients with known hepatocellular carcinoma (HCC). Hepatocellular carcinoma is cancer of the liver.

Hepatocellular carcinoma is estimated to affect approximately 35,000 U.S. patients in 2013. Lipiodol is currently under FDA evaluation for safety and efficacy with a proposed indication “for selective hepatic intraarterial use in computed tomography of the liver to visualize and localize lesions in adults with known HCC.”

3. Treatment of Acute Radiation Syndrome

Soligenix, Inc. announced on Monday, September 16, 2013, that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) granted orphan drug designation to the active ingredient SGX94 for the treatment of acute radiation syndrome (ARS). SGX94 is an innate defense regulator (IDR), a new class of short, synthetic peptides that accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma, radiation and/or chemotherapy.

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