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

ORPHAN DRUG COMMERCIAL CEILING OF $ 25 MIL. ANNUAL SALES is included in a proposal being floated for comment by Rep. Waxman (D-Calif.). Proposed as a potential substitute for his Orphan Drug Amendments (HR 3459), passed by the House Commerce Committee in October, the draft would redefine orphan products as those with a total annual market value of less than $ 25 mil. A summary explains that if the market value, which includes "all sponsors and all orphan uses," exceeds the threshold within any 12-month period, then the product's market exclusivity is terminated. Currently, orphan designations are available for drugs to treat conditions occurring in less than 200,000 patients. Because the legislation also proposes to permit multiple, "simultaneous" developers of an orphan product to share exclusive markets, FDA would be required to "aggregate the sale of all" sponsors of a given orphan drug. Consequently, each sponsor would be required under the proposal to report orphan product sales to FDA when they reach $ 15 mil., and the "report shall be certified by an independent accountant." Waxman's proposal would meet resistance from PMA and possibly FDA. The agency historically has been averse to monitoring sales records, and PMA, which to date has been neutral on Waxman's bill, reportedly will vigorously oppose provisions tied to a cap on sales. A "simultaneous development" section of the proposal would permit FDA to approve subsequent products on the market during a first orphan drug's exclusivity period if the subsequent sponsors are "eligible" and file full, not abbreviated, marketing applications. The proposal defines an eligible company as one that files for an IND exemption before the later of two events: the first submission of an NDA after orphan status has been granted or the first designation of the orphan drug after an NDA has been submitted. The provision encourages first developers to request orphan designation as early in the review process as possible. To be considered eligible to share market exclusivity as a simultaneous developer, a company "must request designation of their drugs, if they are not already designated, within 60 days of becoming eligible," according to the summary. The intent of this provision is to notify "all other parties of the presence of an eligible" company.

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

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

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