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GlycoMimetics' Rivipansel Gets Rare Pediatric Disease Status

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GlycoMimetics, Inc. ((GLYC - Free Report) ) announced that the FDA has granted a Rare Pediatric Disease designation for its lead candidate rivipansel for the treatment of sickle cell disease (“SCD”) in patients 18 years old and younger. Sickle cell disease is a group of blood disorders typically inherited from a person's parents.

Shares of the company have decreased 38.2% year to date compared with the industry’s 7.6% decline.


Rivipansel is a glycomimetic drug candidate and the company plans to release full data set from the phase III RESET program at upcoming medical meetings.

The Rare Pediatric Disease designation makes the candidate eligible for a Rare Pediatric Disease Priority Review Voucher (“PRV”) upon approval. A PRV allows the holder of the voucher to receive an expedited six-month review from the FDA for new drug application or biologics license application instead of the usual ten-month review. Priority review vouchers are also awarded by the FDA for the development of treatments for certain tropical diseases and medical countermeasures.

There are some companies that are developing or have developed candidates for the treatment of SCD. bluebird (BLUE - Free Report) is developing lentiGlobin gene therapy for adult and adolescent patients with SCD. Global Blood Therapeutics’ (GBT - Free Report) Oxbryta (voxelotor) — an oral, once-daily treatment — is marketed for the treatment of SCD. Novartis (NVS - Free Report) has Adakveo (crizanlizumab) in its portfolio to reduce the frequency of vaso-occlusive crises or pain crises in patients aged 16 years or above with SCD. 

GlycoMimetics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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