Orphan drugs current status

Currently, there are a number of systemic and intestine-selective MTP inhibitors, including lomitapide (23, BMS-201038, AEGR-733), implitapide (24), JTT-130, SLx-4090, and R-256918 (latter three structures not disclosed) believed to be in active development [60]. In a meta-analysis of three Phase II clinical trials, lomitapide as monotherapy or in combination with ezetimibe, atorvastatin, or fenofibrate significantly reduced LDL cholesterol (up to 35% as monotherapy and 66% in combination with atorvastatin) and was well tolerated with less than 2% discontinuation due to abnormal liver function [61]. Lomitapide has also been granted orphan drug status for the treatment of homozygous familial hypercholesterolemia [59]. Results of a Phase II study of JTT-130 for type 2 diabetes are expected in August 2010 [59,60]. 

Asbury, C.H. Evolution and current status of the orphan drug act. Intern. J. Technol. Assess. Health Care 1992,... 

Asbury, C.H. Evolution and Current Status of the Orphan Drug Act. Intern. J. Technol. Assess. Health Care 1992, 8 (4),. 573-582. 47. Arno, P.S. Bonuck, K. Davis, M. Rare Diseases, Drug Development, and AIDS The Impact of the Orphan Drug Act. Milbank Q. 1995, 73 (2), 241-252. 

The Orphan Drug Act of 1983 (Public Law 97-414) provided a 50-percent tax credit for qualifying clinical R D on investigational drugs that have been granted orphan status by the FDA. The credit is available only for qualifying clinical research, not for animal or laboratory research and not for supervisory or other kinds of R D expenditures typically disallowed by the Internal Revenue Service. Also, when the credit is applied, the expenses cannot be deducted, so the net cost of a dollar of qualifying research under this credit is effectively 0.50. Companies without current taxable income cannot save the credit for use in future years, however, so startup research-based firms may not have access to this credit. 

A. Parenteral (United States). Hydroxocobalamin, 1 mg/mL IM 10- and 30-mL vials (may contain parabens). The United States does not have a formulation practical to treat acute cyanide poisoning (because of preparation time and the large fluid volume required). Orphan-USA has obtained orphan drug status for hydroxocobalamin Cyanokit with no current plans to move it to market. 

Genentech subsequently challenged the FDA s decisions in court by arguing the FDA did not have the authority to grant orphan status to Eli Lilly. The courts ruled against Genentech. Currently, each manufacturer has orphan status for its version of HGH, and each drug is sold on the market. 37... 

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