Updates on the regulatory front are eagerly awaited by investors in the pharma/biotech sector as they impact the share price of the concerned company. Regulatory updates include events like filing of marketing applications, acceptance of these applications for review by the regulatory agencies, reviews by advisory panels and finally, a response from the regulatory agency regarding the approval status.
Late last week, Horizon Pharma plc (HZNP) announced that the FDA has granted orphan drug status to Actimmune for the treatment of Friedreich's ataxia (FA). According to Horizon Pharma, FA is a degenerative neuro-muscular genetic disorder affecting approximately one in every 50,000 people in the U.S.
Under the U.S. Orphan Drug Act, a drug or biological product is granted the special status if it is considered capable of treating a rare disease or condition that affects less than 200,000 patients in the U.S. On receiving orphan drug designation for a candidate, the company developing it is entitled to various incentives, including tax credits for qualified clinical testing. Moreover, the Prescription Drug User Fee Act (PDUFA) fees are waived off the marketing application for an orphan drug designated candidate. Additionally, this status should make Actimmune eligible for seven years of market exclusivity for the treatment of FA, on approval.
We note that Actimmune, added to Horizon Pharma’s portfolio following the acquisition of Vidara Therapeutics, is already approved in two indications – reduction of the frequency and severity of serious infections associated with chronic granulomatous disease and slowing down the deterioration of severe, malignant osteopetrosis.
The orphan drug status for Actimmune is important for Horizon Pharma. This designation should boost the development program on Actimmune for FA. As per the company’s press release, there are no treatments currently approved in this indication. This underscores an unmet market need for newer therapies.
Last month, Prana Biotechnology’s (PRAN) PBT2 received orphan drug designation in the U.S. for the treatment of Huntington disease, a neurodegenerative genetic disorder.
Currently, Horizon Pharma carries a Zacks Rank #3 (Hold). Other well-placed stocks in the health care sector are Mallinckrodt plc (MNK) and Actavis plc (ACT). Both carry a Zacks Rank #1 (Strong Buy).
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