Ultragenyx Pharmaceutical Inc (NASDAQ:RARE)’s CEO Dr. Emil Kakkis has claimed that he is on the brink of getting a huge breakthrough in the pediatric studies that will lead to the production of a drug that will be the cure of life threatening bone diseases. During his intervention on CNBC, Kakkis analyzed the bone disorders in details and said that his company was treating adults in the bone diseases. He now thinks that Pediatric patients are mostly children and they have the most bone diseases, mainly because they don’t get to produce enough phosphate that is necessary to keep the bones working
“Children have the most bone diseases, they need the most phosphates, you don’t have phosphate, you can’t make bone,” said Dr. Emil Kakkis
Explaining the need of the drug to fight the threatening bone disease, Kakkis said that the deformed bones lead to a number of surgeries that practically dilute the chances of survival and if one gets lucky enough to do so, he gets disabled for the rest of his life. He added that Phase 2 of the pediatric trials will start this year, whereas, the Phase 3, which is the most important in finalizing the feasibility of this drug, will kick off in 2015.
Answering the question of pricing, CEO of Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) said that his company has not set the price of this drug yet as they are still working on it. He didn’t comment on the rumors that this drug will cost $100,000.
“We haven’t set pricing, we don’t know how well the drug works yet, it’s something we would do much later on but at this point we think this treatment has the potential to be life changing” said Kakkis. Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) will definitely come in the limelight if they get successful in launching this drug.