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ORPHAN PRODUCT NDAs/PLAs PENDING TOTAL 40

Executive Summary

ORPHAN PRODUCT NDAs/PLAs PENDING TOTAL 40 with an additional 133 orphan drugs in clinical development, according to a survey by the Pharmaceutical Manufacturers Association released Sept. 12. PMA released the report in cooperation with FDA's Office of Orphan Products Development and the National Organization for Rare Disorders. Because some drugs are being investigated for more than one disease, the 133 products described in the survey represent a total of 172 orphan designations with treatment potential for at least 93 rare disorders. PMA members and their research affiliates account for 89 of the orphan designations; non-PMA companies represent 70 more, and other research scientists and organizations are responsible for 13. According to PMA's data, Bristol-Myers and Schering-Plough have been the most active companies in obtaining orphan designations, with 12 and 11, respectively. However, Johnson & Johnson subsidiary Ortho had the most NDAs and PLAs submitted for orphan products: four of Ortho's five designated orphans are currently being reviewed by FDA. These include the LHRH product Lutrepulse for induction of ovulation, Eprex (erythropoietin) for anemias associated with HIV-infection and end-stage renal disease, and histrelin for treatment of precocious puberty. Bristol-Myers has three NDAs on file that are orphan drugs -- Mucomyst 10 I.V. (acetylcysteine), for acetaminophen overdose, and d,1-sotalol HCl, for treatment and prevention of life-threatening ventricular tacharrhythmias. Schering-Plough has filed for only one of its orphans, Intron A (recombinant alfa-2b interferon), for bladder carcinoma. Companies responding to a survey for the National Commission on Orphan Diseases reported investing $ 54.6 mil in rare disease R&D, with 94.5% of that total coming from PMA companies, according to PMA President Mossinghoff. Mossinghoff addressed recent movements to change the exclusivity incentives of the Orphan Drug Act. "I would be concerned that if (BRACKET)the exclusivity provisions(BRACKET) were changed, that that would simply be viewed as a change in the ground rules, and I think it would have a serious effect on the amount of PMA company resources that would be devoted to orphan drug research," he said.
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