The priority review voucher that BioMarin Pharmaceutical Inc. received for Vimizim (elosulfase alfa) and recently sold to Sanofi and Regeneron Pharmaceuticals Inc. for $67.5 million appears to have been almost an afterthought and was helped by the agency’s liberal interpretation of the statutory language creating the voucher program.

BioMarin submitted a pediatric voucher request on Jan. 7, 2014, less than six weeks before the Morquio A syndrome treatment’s approval on Feb. 14, 2014, and more than nine months after the BLA originally was submitted.

Vimizim’s indication in the adult population “is merely a continuum of the pediatric indication, and does not represent a different adult indication,” ODE III Acting Deputy Director Egan said.

In fact, FDA received the voucher request during the three-month extension in the user fee goal date. The original user fee date was in November 2013 but the review was extended due to a major CMC amendment.

FDA previously has said that since the pediatric voucher program is relatively new and formal guidance has not yet been issued, it has tried to promote awareness of the program by informing sponsors of potential eligibility Also see "BioMarin Tests FDASIA’s Pediatric Voucher Waters With Vimizim Approval" - Pink Sheet, 24 February, 2014..

In response to questions about the timing of the pediatric voucher request, BioMarin told “The Pink Sheet”: “During the application process, we worked with the FDA and followed the agency’s direction on submitting an application. We don’t discuss the details of the dialogue between the company and the FDA during the BLA review process.”

Pediatric, Adult Indications Inseparable

Under the FDA Safety and Innovation Act provisions that established the rare pediatric disease voucher program, a product can be eligible for a voucher only if it “does not seek approval for an adult indication in the original rare pediatric disease application.”

BioMarin received the voucher for Vimizim even though the enzyme replacement therapy’s indication is not limited to pediatric patients. Rather, Vimizim carries a broad claim for patients with Morquio A syndrome, also known as Mucopolysaccharidosis type IVA.

FDA review documents indicate the agency’s Office of Orphan Products Development (OOPD) took a liberal view in applying this statutory provision to Vimizim – news that should buoy other sponsors of rare pediatric disease treatments.

In a Feb. 14 memo, Office of Drug Evaluation III Acting Deputy Director Amy Egan references a Jan. 14 memo in which OOPD “opined that MPS IVA meets the FDASIA definition of a rare pediatric disease. FDA has further determined that the Vimizim BLA submission represents a rare pediatric disease product application.”

“While Vimizim’s indication encompasses an adult population, it is understood that the indication in the adult population is merely a continuum of the pediatric indication, and does not represent a different adult indication. Therefore, a priority review voucher will be granted at the time of approval,” Egan said.

What The Money Means To BioMarin

For BioMarin, the voucher request submitted late in the review process and FDA’s liberal interpretation of the statutory provisions meant a $67.5 million payoff with the Sanofi/Regeneron deal, which amounts to a $50 million after-tax benefit Also see "How Much Is A Priority Review Worth? $67.5 Million, Sanofi/Regeneron Say" - Pink Sheet, 11 August, 2014..

“Leveraging the sale of the priority review voucher to reinvest in products to treat rare and ultra-rare diseases makes the most sense for BioMarin given our stage of growth,” CEO Jean-Jacques Bienaimé said in a press release. “We are very pleased that a patient population beyond BioMarin’s will potentially be able to benefit from a faster drug application review process.”

When asked what the company planned to do with the voucher sale proceeds, a spokeswoman said the money gives BioMarin additional flexibility and “we are considering many potential uses for the proceeds that will support our growth.”

Among the possibilities, she said, are expanding BioMarin’s San Rafael, Calif., facility, which houses the company’s corporate headquarters and related operating activities.

In March, BioMarin completed the acquisition of a multi-building commercial property in San Rafael where it had been leasing space. The acquisition was aimed at enabling future expansion of the corporate headquarters to accommodate anticipated headcount growth, BioMarin reported in its second quarter 10-Q filing with the Securities and Exchange Commission.

The spokeswoman also said BioMarin could use the proceeds to build out a manufacturing facility in Shanbally, Ireland.

That facility was purchased in 2011 but has not yet been approved by FDA or the European Medicines Agency. “We intend to make a substantial investment in the build-out of the Shanbally facility in order to manufacture Vimizim and other products,” the SEC filing states.

Vimizim Voucher A Result Of BioMarin’s Late Request, FDA Flexibility

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