Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

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

UsernamePublicRestriction

GENETICALLY ENGINEERED PRODUCTS MAKE UP NEARLY ONE-THIRD OF FDA ORPHAN DRUG DESIGNATIONS IN 1987; FDA DESIGNATES 29 ORPHANS

Executive Summary

FDA granted orphan drug/biologic status to nine genetically engineered products in the first nine months of 1987, according to a recently published list of FDA orphan product designations. The nine genetically engineered products account for almost one-third of FDA's 29 orphan designations during the period. Among the nine genetically engineered orphan products are Xoma's monoclonal antibody linked immunotoxins XomaZyme-791, in Phase I studies for treatment of metastatic colorectal cancer adenocarcinoma, and XomaZyme-H65, in Phase II trials for in vivo treatment of graft v. host disease and/or rejection in patients who have received bone marrow transplants. Xoma has been one of the most active firms in seeking orphan drug status for its products, with three designations granted in 1985 and one in 1986. The firm's other orphan products include the septic shock monoclonal antibody Xomen-E5, the imaging agent XomaScan-Mel, the melanoma immunotoxin XomaZyme-Mel, and a transplant immunotoxin. Recombinant human growth hormone and erythropoietin, two products with large revenue potential, were also among the biotech products designated as orphans during the nine month period, with five firms receiving orphan status. Genentech's Protropin II, Serono's Saizen, and Nordisk's Norditropin were designated as orphans for treatment of short-stature children with growth hormone deficiency. Lilly, which currently has exclusivity for its methionyl-free growth hormone approved in March, received orphan drug status for Humatrope in 1986. Norditropin's recent orphan designation was also granted for adjunctive use in the induction of ovulation in women with certain types of infertility. Ortho, a marketing partner for Amgen's recombinant erythropoietin, and McDonnell Douglas, both received orphan status for erythropoietin as a treatment for anemia associated with end-stage renal disease. Under its licensing agreement with Amgen, Ortho has rights to the U.S. EPO market for pre-dialysis anemia patients and other non-dialysis anemia indications. Amgen has exclusive rights to patients being treated with dialysis. Amgen recently finished Phase III studies with its recombinant product and is planningan NDA submission before year-end. Schering's recombinant alfa interferon (Intron A) received orpan status for the treatment of chronic myelogenous leukemia, metastatic renal cell carcinoma, AIDS-related Kaposi's Sarcoma and ovarian carcinoma. The product, approved in June 1986 for hairy cell leukemia, is among several products on the January-September list that are already on the market for other indications. Other marketed products with new orphan indications include Elkins-Sinn's Duramorph (preservative-free morphine sulfate), currently approved as an analgesic, and KabiVitrum's Cyklokapron (tranexamic acid), approved in December 1986 for prevention of hemorrhage in hemophiliac tooth extractions. Duramorph received orphan status for administration in microinfusion devices in repeated doses or constant infusion, for epidural use, and for intrathecal use. Cyklokapron's orphan designation is for treatment of patients undergoing prostatectomy where there is hemorrhage or risk of hemorrhage. Bristol-Myer's Mucomyst I.V. received orphan status for acetaminophen overdose. Only the oral form of Mucomyst currently carries the overdose indication. Eight of the new orphan drug designations are for the treatment or diagnosis of cancer. In addition to Intron A and XomaZyme-Mel, cancer-related orphans include: NeoRx's murine monoclonal antibody kit for imaging malignant melanoma metastases; Sanofi's ST1-RTA immunotoxin for treatment of patients with B-chronic lymphocytic leukemia, which is also designated for graft v. host disease; and Pharmachemie B.V.'s 5-AZA-2'-deoxycytidine (DAC) for acute leukemia. Warner-Lambert's pentostatin, Bristol-Myer's ifosfamide, and Lederle's mitoxantrone (Novantrone) are also among the products receiving orphan status for cancer indications. The National Cancer Institute is considering Treatment IND status for the orphan indications granted to ifosfamide and pentostatin. ("The Pink Sheet" Oct. 5, T&G-11). NCI is also seeking Treatment IND status for Novantrone, designated an orphan for acute non-lymphocytic leukemia, as therapy for advanced breast cancer. Charts omitted.

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

Latest Headlines
See All
UsernamePublicRestriction

Register

PS012695

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

Cancel