Imara receives rare paediatric disease designation from FDA for lead product candidate IMR-687 for sickle cell disease

Imara

15 May 2017 - Imara announced today that the U.S. FDA has granted rare paediatric disease designation to IMR-687, the company’s lead product candidate. 

IMR-687 is the first SCD candidate to be designated as a drug for a rare paediatric disease, and this designation builds upon, and is complimentary to, the FDA’s earlier grant of orphan designation.

Imara is conducting a Phase 1a clinical study to evaluate the safety and pharmacokinetics of IMR-687 in healthy volunteers. In addition, Imara will assess pharmacodynamic markers. Pending the positive outcome of this Phase 1 study this summer, Imara will initiate a Phase 2a study in adult patients living with sickle cell disease later this year and then expects to initiate the Phase 2 in paediatric patients in 2018.

Read Imara press release

Michael Wonder

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Michael Wonder

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US , Orphan drug , Submission