variant protein targeted drug
Defined in 1 place of the United States Code.
the term “variant protein targeted drug” means a drug that—
(A) is the subject of an application under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] for the treatment of a rare disease or condition (as such term is defined in section 360bb of this title) that is serious or life-threatening;
(B) modulates the function of a product of a mutated gene where such mutation is responsible in whole or in part for a given disease or condition; and
(C) is intended to treat one or more patient subgroups, including subgroups of patients with different mutations of a gene, with the same disease or condition.
Source
- 2026–present21 U.S.C. § 360ff-1Food and Drugs · Targeted drugs for rare diseases · for purposes of this section
the term "variant protein targeted drug" means a drug that—
(A) is the subject of an application under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] for the treatment of a rare disease or condition (as such term is defined in section 360bb of this title) that is serious or life-threatening;
(B) modulates the function of a product of a mutated gene where such mutation is responsible in whole or in part for a given disease or condition; and
(C) is intended to treat one or more patient subgroups, including subgroups of patients with different mutations of a gene, with the same disease or condition.
Nothing in this section shall be construed to—
Source
- 2016–202621 U.S.C. § 360ff-1Food and Drugs · Targeted drugs for rare diseases · for purposes of this section