Company

Product

Description

Indication

Status

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Patisiran

RNAi therapeutic

Hereditary transthyretin-mediated amyloidosis with polyneuropathy

Filed marketing application with Brazilian regulatory agency ANVISA, which granted priority review; decision expected in first half of 2020

Boehringer Ingelheim GmbH, of Ingelheim, Germany

Ofev (nintedanib)

Antifibrotic

Chronic fibrosing interstitial lung diseases with progressive phenotype

FDA granted breakthrough therapy designation; application under review

Frequency Therapeutics Inc., of Woburn, Mass.

FX-322

Combination of small-molecule drugs designed to activate inner ear progenitor cells to induce hair cell regeneration

Sensorineural hearing loss

FDA granted fast track designation

Eicosis LLC, of David, Calif.

EC-5026

Small-molecule inhibitor of epoxide hydrolase

Pain

FDA cleared the IND for phase I trials

Emergent Biosolutions Inc., of Gaithersburg, Md.

CHIKV VLP

Virus-like particle vaccine

Chikungunya virus

Granted Priority Medicines, or PRIME, designation by EMA’s Committee for Medicinal Products for Human Use

Krystal Biotech Inc., of Pittsburgh

KB-105

Gene therapy

Transglutaminase-1-deficient autosomal recessive congenital ichthyosis

EMA’s Committee for Orphan Medicinal Products issued a positive opinion for orphan designation

Motif Bio plc, of London

Iclaprim

Selective bacterial dihydrofolate reductase inhibitor

Hospital-acquired bacterial pneumonia, including ventilator-associated bacterial pneumonia

Received minutes of type B meeting with FDA, confirming single well-designed and well-controlled phase III trial demonstrating efficacy and safety, along with data on potential mechanism of hepatic injury, would enable submission of NDA; company said partner or other entity with lower cost of capital would be needed


Notes

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