SAN RAFAEL, Calif.,
Nov. 27, 2017 /PRNewswire/ --
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it
has entered into a definitive agreement to sell the Rare Pediatric
Disease Priority Review Voucher (PRV) it obtained in April of this
year for a lump sum payment of $125,000,000. The Company received the
voucher under a U.S. Food and Drug Administration (FDA) program
intended to encourage the development of treatments for rare
pediatric diseases. BioMarin was awarded the voucher when it
received approval of Brineura®, a new biological product for
patients with late infantile neuronal ceroid lipofuscinosis type 2
(CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, a
form of Batten disease. The transaction remains subject to
customary closing conditions, including anti-trust review.
"We are proud to be able to participate in a program that
encourages investment in the development of therapies for children
with rare diseases. BioMarin will direct the proceeds from
this voucher sale towards additional investment in an already
robust pipeline of products to treat rare and ultra-rare diseases,"
said Jean-Jacques Bienaimé, Chief Executive Officer of
BioMarin. "We are very pleased that this voucher will be
accelerating the availability of a therapy for patients."
This is the second PRV that BioMarin has sold. In
July 2014, BioMarin received
$67.5 million from Regeneron Ireland,
an indirect, wholly-owned subsidiary of Regeneron Pharmaceuticals,
Inc., in exchange for a voucher awarded when it received approval
of Vimizim® for patients with the rare disease,
Mucopolysaccharidosis type IVA, also known as Morquio A
syndrome.
Pediatric Disease Priority Review Voucher Sale Impact to
BioMarin Financial Guidance
The sale of the PRV will be recorded as a $125 million gain on sale of an intangible asset
and will also be associated with approximately $25 million of income tax expense. As a
result of the sale, the Company will update its GAAP Net Loss
guidance by the $100 million net
after tax gain, and for full-year 2017, the GAAP Net Loss guidance
will be reduced to between $(10) million and
$(30) million. The sale of the PRV is a special item
that will be excluded from Non-GAAP Income and consequently the
Non-GAAP Income guidance for the full-year 2017 is unchanged at
$60 to $80 million.
About the Pediatric Disease Priority Review Voucher
Program
The PRV is issued to the sponsor of a rare pediatric disease
product application that entitles the holder to priority review of
a single New Drug Application or Biologics License
Application. The sponsor receives the voucher upon approval of
the rare pediatric disease product application. PRVs may be
sold or transferred, and there is no limit on the number of times a
priority review voucher can be transferred.
About Food and Drug Administration Standard Review and
Priority Review Designations
Prior to approval, each drug marketed in the United States must go through a detailed
FDA review process. In 1992, under the Prescription Drug User
Act (PDUFA), FDA agreed to specific goals for improving the drug
review time and created a two-tiered system of review times -
Standard Review and Priority Review. Standard Review can be
accomplished in a ten-month time frame from the time the
application is filed by the FDA, which typically occurs
approximately 60-days following submission of the
application. A Priority Review designation is given to drugs
that offer major advances in treatment, or provide a treatment
where no adequate therapy exists. The FDA goal for reviewing a drug
with Priority Review status is six months from the time the
application is filed by the FDA.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for people with serious and
life-threatening rare disorders. The company's portfolio consists
of six commercialized products and multiple clinical and
pre-clinical product candidates.
For additional information, please visit www.biomarin.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
BioMarin®, Brineura® and Vimizim® are registered trademarks of
BioMarin Pharmaceutical Inc., or its affiliates.
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Traci
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Debra
Charlesworth
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BioMarin Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
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(415)
455-7558
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(415)
455-7451
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SOURCE BioMarin Pharmaceutical Inc.