BRIEF—Brazil rejects JCR Pharma's Hunter syndrome therapy

JCR Pharmaceutical's Hunter syndrome treatment pabinafusp alfa (JR141) failed to get the manufacturing and marketing approval in Brazil, according to a statement.

The Brazilian Health Surveillance Agency said the candidate, a fusion protein using JCR's proprietary blood-brain barrier penetrating technology, used a substrate biomarker that lacks the "scientific and clinical validation" to prove clinical improvement with the reduction of the biomarker.

No safety concern was raised. JCR plans to re-apply for the approval with data from its ongoing Phase III global studies.

The treatment is currently sold in Japan under the name Izcargo.