Positive FDA Panel Vote Could Be Coming For BioMarin Pharma, Research Says

• Shares of BioMarin Pharmaceutical Inc. BMRN have appreciated 18.14 percent year-to-date, from $90.28 on January 6.
• Jefferies’ Eun K. Yang has maintained a Buy rating on the company, with a price target of $166.
• Discussions with two additional regulatory experts suggest that the upcoming FDA panel on Kyndrisa could have a positive outcome.

Analyst Eun Yang mentioned that a positive vote by the FDA panel might “not only provide upside, but also increased confidence in 3 additional candidates in Ph1/2, potentially driving BMRN even higher.”

Discussions with two regulatory consultants suggest that there is a high likelihood of positive votes by the FDA AdComm for Kyndrisa on November 24, with approval possibly on December 27.

Yang explained that PDA panel opinions in recent times have been based 75 percent on date and 25 percent on community demand or the debilitating nature of the disease. FDA’s current perspective on Duchenne Muscular Dystrophy (DMD), which Kyndrisa addresses, is to help/approve drugs.

“FDA decision will be based on how compelling the data is & what the discrepancy is in the data,” Yang stated, while noting that since there was no treatment for DMD and given Kyndrisa’s favorable risk-benefit profile, experts appear confident that it will receive FDA approval.

However, experts also believe that a post-marketing study might be required for the drug, unless there is “clearly convincing data in an extension study for Kyndrisa.”