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EMA committee recommends orphan drug designation for Sangamo's genome editing treatments for MPS I & MPS II
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Richmond, California
May 31 , 2019
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Sangamo Therapeutics has announced that the Committee for Orphan
Medicinal Products of the European Medicines Agency (EMA) has issued a
positive opinion on the application for orphan medicinal product
designation (OMPD) for SB-318 and SB-913, Sangamo's genome editing
product candidates for the treatment of rare lysosomal storage disorders
Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively.
The
EMA's OMPD is granted to medicines intended for the treatment,
prevention or diagnosis of life-threatening or chronically debilitating
conditions that are rare and affect less than five in 10,000 persons in
the European Union (EU). The designation provides incentives to advance
the development and commercialization of orphan medicines, which include
access to the EU centralized authorization procedure and potential for
market exclusivity for a period of up to ten years.
MPS I and
MPS II are caused by mutations in the genes encoding alpha-L-iduronidase
(IDUA) and iduronate 2-sulfatase (IDS) enzymes, respectively. Using
Sangamo's zinc finger nuclease (ZFN) genome editing technology, SB-318
(for MPS I) and SB-913 (for MPS II) are designed as a single treatment
strategy intended to provide stable, continuous production of the IDUA
or IDS enzyme for the lifetime of the patient.
SB-318 and SB-913
have already received Orphan Drug, Fast Track and Rare Pediatric
Disease designations from the U.S. Food and Drug Administration (FDA).
The phase 1/2 clinical trials for these programs, evaluating SB-318 and
SB-913 in adults with MPS I and MPS II, respectively, are open and
enrolling subjects.
Sangamo's ZFN-mediated in vivo genome
editing approach makes use of the endogenous albumin gene locus, a
highly expressing and liver-specific site that can be edited with ZFNs
to accept and express therapeutic genes. The approach is designed to
enable the patient's liver to permanently produce circulating
therapeutic levels of a corrective protein. The ability to permanently
integrate the therapeutic gene in a highly specific, targeted fashion
significantly differentiates Sangamo's in vivo genome editing approach
from conventional AAV cDNA gene therapy. The design of these programs is
ultimately to target a population that includes pediatric patients, and
it will be important in this population to be able to produce stable
levels of therapeutic protein for the lifetime of the patient.
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TOPICS
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India needs to align with global good practices to reinvogorate its capability in pharmaceutical manufacture. The regula ...
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