BioMarin could cost $300,000.
By Raif Karerat
WASHINGTON, DC: An experimental drug that was designed to treat dwarfism by lengthening children’s bones is being met with a certain amount of measured vitriol from both parents and the leading U.S. group that represents those with dwarfism, the Little People of America.
Leaders in the organization are in regular contact with San Rafael, California-based BioMarin Pharmaceutical Inc., according to Bloomberg; they worry the drug is being presented as a way to address an “affliction.”
Parents like Chelley Martinka, whose daughter has the condition, achondroplasia, say they have no intention of giving their children injections of BioMarin’s BMN 111.
“My daughter is incredibly smart, she’s funny, she is the most loving person I’ve ever met,” Martinka, who doesn’t have achondroplasia, told Bloomberg. “I can’t ask for anything else. To do something superficial, to give her a couple of inches, it’s ridiculous.”
BioMarin’s goal is to persuade parents of the drug’s potential to add height and avert health issues that come with the genetic disorder, such as neurological and breathing problems that can occur when he spinal cord, tonsils and other unaffected tissue grows faster than the surrounding bone, said Melita Irving, the lead investigator of BioMarin’s trial and a consultant in clinical genetics at the Guy’s and St. Thomas’ hospital system in London.
“If it’s proven to be safe, I don’t think any average-height person with a baby is going to say no,” said Kristina Gray, the mother of a 4-year-old boy with achondroplasia in Warwickshire, England. “I see this as giving my son a better quality of life.”
Michael Yee, an analyst at RBC Capital Markets, estimates that BioMarin could charge $300,000 for the drug, which would make it the company’s biggest product.
Reuters reported the drug could rake in sales of about $700 million worldwide, according to a note Raluca Pancratov of SunTrust Robinson Humphrey wrote to his clients.
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