New Medicines

Mucopolysaccharidosis I (MPS I - Hurler syndrome)


New molecular entity

Development and Regulatory status

Phase II Clinical Trials
Jun 18RGX-111 has received Fast Track designation from the FDA [1,2].
Oct 15RGX-111 has received orphan drug and rare paediatric disease status from the FDA [1,2].


Designed to deliver the gene encoding the IDUA enzyme using the adeno-associated virus serotype 9 vector, which has the ability to traverse the blood-brain barrier to target the central nervous system. It is given as a single injection cerebrospinal fluid
Mucopolysaccharidosis type I affects males and females in equal numbers, with an incidence of about 1 in 100,000 live births for the severe type, and an incidence of about 1 in 500,000 live births for the attenuated type [3]
Mucopolysaccharidosis I (MPS I - Hurler syndrome)

Trial or other data

Feb 22Company presents positive initial data of NCT03580083. RGX-111 is reported to be well-tolerated in the five pts enrolled in the PI/II clinical trial with no drug-related serious adverse events. One pt in Cohort 1 has completed the 48-week immunosuppression regimen per the study protocol [5].
Apr 19Company initiates PI/II multicenter, open-label trial to evaluate the safety, tolerability, and pharmacodynamics of intracisternal RGX-111 in pts with Mucopolysaccharidosis Type I (NCT03580083, n=11 aged 4 months and older). The pts are in two dosing groups- 1x10^10 gc/g brain mass or 5x10^10 gc/g brain mass of RGX-111. Study location includes US and Brazil. Primary outcome measure includes number of pts with treatment-related adverse events and serious adverse events at week 24. Estimated primary completion is Oct 22 [4].