Today’s Research Reports on Stocks to Watch: Ultragenyx Pharmaceutical and BioMarin Pharmaceutical
NEW YORK, NY / ACCESSWIRE / May 29, 2018 / Ultragenyx Pharma shares jumped after announcing its clinical trial results for Crysvita® ( burosumab), while BioMarin Pharma earned approval for its Palynziq™ ( pegvaliase- pqpz) Injection.
RDI Initiates Coverage on:
Ultragenyx Pharmaceutical Inc.
https://rdinvesting.com/news/?ticker=RARE
BioMarin Pharmaceutical Inc.
https://rdinvesting.com/news/?ticker=BMRN
Shares of Ultragenyx Pharmaceutical increased 4.36 percent to close at 67.31 on Friday, following its announcement of pediatric Phase 2 clinical trial of Crysvita® ( burosumab) results for the treatment of X-linked hypophosphatemia (XLH) in children aged 5 to 12 years. XLH is a rare, chronic progressive musculoskeletal disorder that affects children and adults. Crysvita is the first treatment to target the underlying pathophysiology of XLH – excess production of fibroblast growth factor 23 (FGF23), a hormone that regulates phosphate excretion and active vitamin D production by the kidney. According to Tom Carpenter, M.D., the lead study investigator, Director of the Yale Center for X-Linked Hypophosphatemia, and Professor of Pediatric Endocrinology at Yale University School of Medicine "This study was the natural extension of recent discoveries related to disease mechanisms in XLH, and in particular FGF23's role as a mediator of renal phosphate metabolism." "The therapeutic approach taken, to directly inhibit FGF23's renal actions, demonstrates the potential advantages to therapy of metabolic disease when targeting a central disease mechanism. Sustained improvements in serum phosphorus resulted, with corresponding improvement in skeletal abnormalities. The approach should prove to be an important advance in the therapy of XLH," he added.
Access RDI's Ultragenyx Pharmaceutical Inc. Research Report at:
https://rdinvesting.com/news/?ticker=RARE
On Friday, the BioMarin Pharmaceutical's stocks gained 4.77 percent to close at 92.49, following its announcement of approval from the U.S. Food and Drug Administration (FDA) for Palynziq™ ( pegvaliase- pqpz) Injection to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. The symptom of PKU is inability to break down Phe, an amino acid that is found in all forms of protein, if left untreated, high levels of Phe may toxicate the brain and might lead to serious neurological and neuropsychiatric-related problems, impacting the way a person thinks, feels, and acts. "BioMarin is thrilled to be able to offer this important new therapy to adults with PKU who are unable to control their Phe levels with existing options. The approval of Palynziq is the culmination of more than a decade of perseverance by BioMarin employees dedicated to bringing treatments to PKU adult patients," said Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin.
Access RDI's BioMarin Pharmaceutical Inc. Research Report at:
https://rdinvesting.com/news/?ticker=BMRN
Our Actionable Research on Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) can be downloaded free of charge at Research Driven Investing.
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