Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By



Executive Summary

Lyphomed's price for Pentam aerosolized pentamidine "does not constitute an abuse of the Orphan Drug Act," National Organization for Rare Disorders Executive Director Abbey Meyers told Sen. Pryor (D-Ark.). In a Dec. 27 letter to the chairman of the Senate Special Committee on Aging, Meyers maintained that the product, sold for prevention of Pneumocystis carinii pneumonia, was "adopted" by Lyphomed and developed "as a public service when only 300 AIDS patients were identified in the U.S., and no one expected the disease to become an epidemic." Meyers' letter was in response to a legislative proposal by the senator that would allow a second manufacturer to share the market exclusivity of an original orphan drug if the second product features a superior drug delivery system ("The Pink Sheet" Dec. 18, p. 3). Pryor's proposal was designed specifically to permit Fisons' Nebupent pentamidine product to share Lyphomed's exclusive market for Pentam on the grounds that the Fisons product uses a more efficient nebulizer and could be used at half the cost of Pentam. Meyer argued that "any AIDS patient who cannot afford pentamidine can get it for free from Lyphomed. Why then should the act be undermined simply to let another company prosper?" Although NORD would oppose any amendments to limit exclusivity, Meyers wrote that the organization would support the exclusion of AIDS drugs from Orphan Drug Act benefits. The law "was not written for an epidemic, and we would support legislation to remove AIDS and its opportunistic infections from the act," she said. * NORD asserts that "only human growth hormone (hGH) fits" the definition of a product that constitutes an abuse of the orphan drug law. The organization cites three reasons: R&D on the product began in 1979, four years before the law's enactment; "Genentech submitted documents to the federal court (in a suit designed to stop approval of Eli Lilly's version of hGH) stating that hGH is not an orphan drug and never should have been designated as an orphan drug"; and hGH is approved to treat only 10,000 cases of pituitary dwarfism but is used by short children with normal pituitary levels. Although hGH's "outrageous" price is "inexcusable," Meyer said, "it is not the price that constitutes an abuse" of the act "but the overall size of the $150 annual market." Fisons reportedly also does not support the Pryor proposal. Nonetheless, the senator favors permitting the marketing of Nebupent due to its price advantage. In an April 18 letter to the National Institutes of Health and a May 5 letter to FDA, Fisons indicated that Nebupent would be priced to patients at $40 or no more than $50 per month. Pentam costs approximately $100 per month. Pryor's proposal has the support of the Sloan-Kettering Cancer Center, which initiated the work on aerosolized pentamidine before either Lyphomed or Fisons became involved in development of a product. Sloan-Kettering "researchers were the first to explore" the use of aerosolized pentamidine to prevent AIDS-related pneumonia, Quirk noted. "We chose an arrangement with Fisons, Inc. because of their superior nebulizer technology and ability to conduct classical clinical trials of the sort being demanded by the FDA." In a Dec. 19 letter to the senator, Senior VP-Research Resources Management James Quirk said "we support your efforts to correct the unintentional inequities of this current Orphan Drug Law. While NORD and the drug industry consider the Orphan Drug Act's exclusivity provisions sacrosanct, fearing that any modifications could discourage future development of orphan products, Sloan-Kettering suggests that institutional research into orphan drugs could be discouraged by the current law and the chance that an opportunity to recoup nearly complete R&D can be delayed for seven years. Lyphomed's exclusivity not only causes "an unnecessary increased cost," but it also "will severely impact future research decisions," Quirk said. Institutions facing "constricting research resources" will "give serious consideration to limiting institutional resources for the support of research programs where the likelihood of transfer of research results to patient application is likely to be limited by potential misuse of the law."

You may also be interested in...

Part D Discount Liability Coming Into Focus: CMS Releases Drug Cost Data

Newly released Medicare Part D data sheds light on the sales hit that branded pharmaceutical manufacturers will face when the coverage gap discount program gets under way in 2011

FDA Skin Infections Guidance Spurs Debate On Endpoint Relevance

FDA appears headed for a showdown with clinicians and the pharmaceutical industry over the proposed new clinical trial endpoints for acute bacterial skin and skin structure infections, the guidance's approach for justifying a non-inferiority margin and proposed changes in the types of patients that should be enrolled in trials

Shire Hopes To Sow Future Deals With $50M Venture Fund

Specialty drug maker Shire has quietly begun scouting deals with a brand-new $50 million venture fund, the latest of several in-house investment arms to launch with their parent company's pipelines, not profits, as the measure of their worth




Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts