Category - Developments

Developments

Zynerba Pharma Zygel gets FDA fast track designation for Fragile X

The FDA granted Zynerba Pharma’s (NASDAQ:ZYNE) Zygel fast track designation for behavioral symptoms associated with Fragile X syndrome. Fragile X syndrome, a rare genetic condition caused by a mutation in the X...

May 6, 2019

Developments

Savara gets FDA fast track designation for autoimmune lung disease

The FDA granted Savara’s (NASDAQ:SVRA) Molgradex fast track designation for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP). aPAP occurs when the body’s immune system attacks granulocyte-macrophage...

May 6, 2019

Developments

Theralase presents Phase 1b NMIBC study at AUA conference

Theralase Technologies’ (TSXV:TLT; OTCQB:TLTFF) presented its Phase 1b clinical study of its anti-cancer therapy (ACT) for non-muscle invasive bladder cancer (NMIBC) at the American Urological Association conference in...

May 6, 2019

Developments

Canopy Growth acquires Germany’s C3 Cannabinoid Compound Co. for €225.9 million

Canopy Growth (TSX:WEED; NYSE:CGC) acquired C3 Cannabinoid Compound Co. for €225.9 million (CDN $342.9-million) in a transformative deal that furthers Canopy’s expansion in Europe with prescription medicines. The...

May 2, 2019

Developments

IntelGenx receives second U.S. patent for oral film technology

IntelGenx (TSXV:IGX; OTCQX:IGXT) received its second patent from the U.S. Patent and Trademark Office, covering design and manufacturing of topical oral films for the local treatment of diseases of the oral mucosa using...

May 1, 2019

Developments

EyePoint presents YUTIQ 36-month follow up Phase 3 data at ARVO

EyePoint Pharmaceuticals (NASDAQ:EYPT) presented 36-month efficacy and safety data supporting its YUTIQ three-year micro-insert for the treatment of chronic non-infectious uveitis affecting the posterior segment of the...

May 1, 2019

Developments

LogicBio’s methylmalonic acidemia candidate gets FDA orphan drug designation

LogicBio Therapeutics’ (NASDAQ:LOGC) investigational pediatric genome editing therapy, LB-001, received FDA orphan drug designation for the treatment of methylmalonic acidemia (MMA). MMA, a rare disease caused by...

April 30, 2019

Developments

AVROBIO’s IND for commercial-scale Fabry disease gene therapy cleared by the FDA

AVROBIO’s (NASDAQ:AVRO) IND for AVR-RD-01 – a gene therapy candidate for the treatment of Fabry disease – has been cleared by the FDA, enabling AVROBIO to use its commercial-scale gene therapy manufacturing system...

April 30, 2019

Developments

Vertex Pharma’s KALYDECO gets FDA approval for use in infants with CF

Vertex Pharma’s (NASDAQ:VRTX) KALYDECO obtained FDA approval for use in infants with cystic fibrosis (CF) between the ages of six and 12 months. KALYDECO is indicated in CF patients who have at least one mutation in the...

April 30, 2019

Developments

MyoKardia’s positive Phase 2 results published in Annals of Internal Medicine

MyoKardia’s (NASDAQ:MYOK) positive Phase 2 trial results of mavacamten for the treatment of obstructive hypertrophic cardiomyopathy (HCM) were recently published in the peer-reviewed journal, Annals of Internal Medicine...

April 30, 2019

Category - Developments

Zynerba Pharma Zygel gets FDA fast track designation for Fragile X

Savara gets FDA fast track designation for autoimmune lung disease

Theralase presents Phase 1b NMIBC study at AUA conference

Canopy Growth acquires Germany’s C3 Cannabinoid Compound Co. for €225.9 million

IntelGenx receives second U.S. patent for oral film technology

EyePoint presents YUTIQ 36-month follow up Phase 3 data at ARVO

LogicBio’s methylmalonic acidemia candidate gets FDA orphan drug designation

AVROBIO’s IND for commercial-scale Fabry disease gene therapy cleared by the FDA

Vertex Pharma’s KALYDECO gets FDA approval for use in infants with CF

MyoKardia’s positive Phase 2 results published in Annals of Internal Medicine

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