BioMarin Receives FDA Approval for VOXZOGO™ (vosoritide) for Injection, Indicated to Increase Linear Growth in Children with Achondroplasia Aged 5 and Up with Open Growth Plates

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SAN RAFAEL, Calif., Nov. 19, 2021 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) as we speak introduced that the U.S. Meals and Drug Administration (FDA) has granted accelerated approval to VOXZOGO™ (vosoritide) for Injection, indicated to extend linear progress in pediatric sufferers with achondroplasia 5 years of age and older with open epiphyses (progress plates). This indication is permitted below accelerated approval primarily based on an enchancment in annualized progress velocity (AGV). Continued approval for this indication could also be contingent upon verification and outline of scientific profit in confirmatory research. To meet this post-marketing requirement, BioMarin intends to make use of the continuing open-label extension research in comparison with out there pure historical past.

Voxzogo is the primary FDA permitted therapy for kids with achondroplasia. In sufferers with achondroplasia, endochondral bone progress, an important course of by which bone tissue is created, is negatively regulated on account of a achieve of operate mutation in fibroblast progress issue receptor 3 gene (FGFR3). Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a brand new class of remedy, which acts as a constructive regulator of the signaling pathway downstream of FGFR3 to advertise endochondral bone progress.

“Voxzogo is a medical first that’s rooted in BioMarin’s deal with molecular genetics and targets the underlying explanation for the situation. Greater than a decade of scientific analysis underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its worth as the primary therapeutic therapy choice for kids with achondroplasia,” mentioned Jean-Jacques Bienaimé, Chairman and Chief Government Officer of BioMarin. “We prolong our gratitude to the group, scientific investigators and the youngsters and their households, who participated and proceed to take part in our complete scientific analysis program as we proceed to analyze the total potential of vosoritide.”

“Achondroplasia is a lifelong genetic situation ensuing from the disordered skeletal structure attributable to impaired endochondral bone progress all through childhood,” mentioned Lynda Polgreen, M.D., an investigator in scientific trials for Voxzogo and an Investigator at The Lundquist Institute at Harbor-UCLA Affiliate Professor at David Geffen College of Drugs – UCLA. “This approval is a crucial milestone representing the primary time that physicians will have the ability to supply a remedy focused on the root explanation for the situation for households of youngsters with achondroplasia aged 5 and older.”

“We applaud the FDA for recognizing the pressing unmet medical want for this progressive situation. As a mother or father of a kid with achondroplasia, I see the provision of remedies that impression bone progress as an essential step ahead,” mentioned Amer Haider Co-Founding father of Rising Stronger, a company with a mission to enhance the standard of medical look after little folks via supporting analysis. The group raises nonprofit donations which are granted to researchers targeted on dwarfism.

Mr. Haider added, “BioMarin continues to help the achondroplasia group and has an extended observe document of advancing the usual of care in uncommon genetic circumstances.”

With this approval, the FDA additionally issued a Uncommon Pediatric Illness Precedence Evaluation Voucher (PRV), which confers precedence evaluate to a subsequent drug utility that will not in any other case qualify for precedence evaluate. The uncommon pediatric illness PRV program is designed to encourage improvement of recent medicine and biologics for the prevention or therapy of uncommon pediatric illnesses.

Voxzogo is anticipated to be out there in the US by mid- to late-December, and BioMarin will start the promotion of Voxzogo instantly.

The approval was primarily based on the outcomes of a worldwide randomized, double-blind, placebo-controlled Part 3 research evaluating the efficacy and security of Voxzogo and the open-label extension of this Part 3 research. The research enrolled 121 kids aged 5 to 14.9 with achondroplasia. Baseline imply AGV within the placebo and Voxzogo teams was 4.06 cm/12 months and 4.26 cm/12 months, respectively. At week 52, the change from baseline in AGV was -0.17 cm/12 months for the placebo handled sufferers and 1.40 cm/12 months for the Voxzogo handled sufferers, leading to a statistically important enchancment in AGV of 1.57 cm/12 months in favor of Voxzogo. After the 52 week double blind, placebo–managed, part 3 research, 58 topics initially randomized to Voxzogo enrolled into an open–label extension. Among the many topics who had two years of observe–up since randomization, the development in AGV was maintained.

In August 2021, the European Fee (EC) permitted Voxzogo. Advertising authorization opinions are in course of in Japan, Brazil, and Australia with potential approvals in these nations in 2022.

Vosoritide Security

Security and efficacy of Voxzogo in sufferers with achondroplasia had been assessed in a single 52–week, multi–middle, randomized, double–blind, placebo–managed, Part 3 research. Transient decreases in blood strain have been noticed with Voxzogo. Within the scientific research, 8 (13%) of 60 sufferers handled with Voxzogo had a complete of 11 occasions of transient decreases in blood strain in comparison with 3 (5%) of 61 sufferers on placebo, over a 52-week therapy interval. Sufferers with important cardiac or vascular illness or on anti-hypertensive drugs had been excluded from the trial. To cut back the danger of a lower in blood strain and related signs (dizziness, fatigue and/or nausea), sufferers must be nicely hydrated and have sufficient meals consumption previous to administration.

The most typical opposed reactions, occurring in better than or equal to five% of sufferers handled with Voxzogo and at a proportion better than placebo within the Part 3 research are injection website reactions (together with redness, itching, swelling, bruising, rash, hives, ache), vomiting, joint ache, decreased blood strain, gastroenteritis, diarrhea, dizziness, ear ache, influenza, fatigue, seasonal allergy, and dry pores and skin. See Vital Security Data under and full Prescribing Data and Affected person Prescribing Data for extra security info.

Complete Medical Improvement Program

Voxzogo continues to be studied in a broad scientific improvement program in achondroplasia, and security and efficacy are being additional evaluated throughout totally different ages and over time. Up to now, 243 kids with achondroplasia from eight nations have been enrolled in seven BioMarin scientific research evaluating the protection and efficacy of vosoritide.

Affected person Help for Accessing Voxzogo

To succeed in a BioMarin RareConnections® case supervisor, please name, toll-free, 1-866-906-6100 or e-mail support@biomarin-rareconnections.com. For extra details about Voxzogo, please go to www.voxzogo.com. For extra info concerning this product, please contact BioMarin Medical Data at medinfo@bmrn.com.

About Achondroplasia

Achondroplasia, the most typical type of skeletal dysplasia resulting in disproportionate brief stature, is characterised by slowing of endochondral bone progress, which leads to disproportionate brief stature and disordered structure within the lengthy bones, backbone, face and base of the cranium. This situation is attributable to a achieve of operate mutation within the fibroblast progress issue receptor 3 gene (FGFR3), a detrimental regulator of bone progress. Greater than 80% of youngsters with achondroplasia have dad and mom of common stature and have the situation as the results of a spontaneous change within the gene.

The worldwide incidence price of achondroplasia is about one in 25,000 stay births. Voxzogo is being studied in kids whose progress plates are nonetheless “open,” sometimes these below 18 years of age. That is roughly 25% of individuals with achondroplasia. In Latin America, the Center East, and most of Asia Pacific, there are at present no licensed medicines for achondroplasia.

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Vital Security Data

What’s VOXZOGO used for?

VOXZOGO is a prescription drugs used to extend linear progress in kids with achondroplasia who’re 5 years of age and older with open progress plates (epiphyses).

It’s not recognized if VOXZOGO is protected and efficient in kids with achondroplasia below 5 years of age.

VOXZOGO is permitted below accelerated approval primarily based on an enchancment in annualized progress velocity. Continued approval could also be contingent upon verification and outline of scientific profit in confirmatory trials.

What’s a very powerful security details about VOXZOGO?

VOXZOGO could trigger critical unwanted effects together with a brief lower in blood strain in some sufferers. To cut back the danger of a lower in blood strain and related signs (dizziness, feeling drained, or nausea), sufferers ought to eat a meal and drink 8 to 10 ounces of fluid inside 1 hour earlier than receiving VOXZOGO.

What are the most typical unwanted effects of VOXZOGO?

The most typical unwanted effects of VOXZOGO embrace injection website reactions (together with redness, itching, swelling, bruising, rash, hives, and injection website ache), vomiting, joint ache, decreased blood strain, and stomachache. These should not all of the doable unwanted effects of VOXZOGO. Ask your healthcare supplier for medical recommendation about unwanted effects, and about any unwanted effects that trouble the affected person or that don’t go away.

How is VOXZOGO taken?

VOXZOGO is taken day by day as an injection given below the pores and skin, administered by a caregiver after a healthcare supplier determines the caregiver is ready to administer VOXZOGO. Don’t attempt to inject VOXZOGO till you’ve got been proven the appropriate manner by your healthcare supplier. VOXZOGO is provided with Directions for Use that describe the steps for getting ready, injecting, and disposing VOXZOGO. Caregivers ought to evaluate the Directions for Use for steering and any time they obtain a refill of VOXZOGO in case any modifications have been made.

Inject VOXZOGO 1 time each day, at about the identical time every day. If a dose of VOXZOGO is missed, it may be given inside 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the following day by day dose as standard.

The dose of VOXZOGO is predicated on physique weight. Your healthcare supplier will regulate the dose primarily based on modifications in weight following common check-ups.

Your healthcare supplier will monitor the affected person’s progress and inform you when to cease taking VOXZOGO in the event that they decide the affected person is now not in a position to develop. Cease administering VOXZOGO if instructed by your healthcare supplier.

What must you inform the physician earlier than or throughout taking VOXZOGO?

Inform your physician about the entire affected person’s medical circumstances together with
- If the affected person has coronary heart illness (cardiac or vascular illness), or if the affected person is on blood strain drugs (anti-hypertensive drugs).
- If the affected person has kidney issues or renal impairment.
- If the affected person is pregnant or plans to change into pregnant. It’s not recognized if VOXZOGO will hurt the unborn child.
- If the affected person is breastfeeding or plans to breastfeed. It’s not recognized if VOXZOGO passes into breast milk.

Inform your physician about the entire medicines the affected person takes, together with prescription and over-the-counter medicines, nutritional vitamins, and natural dietary supplements.

Chances are you’ll report unwanted effects to BioMarin at 1-866-906-6100. You might be inspired to report detrimental unwanted effects of prescribed drugs to the FDA. Go to www.fda.gov/medwatch, or name 1-800-FDA-1088.

Please see further security info within the full Prescribing Information and Patient Information.

About BioMarin

BioMarin is a worldwide biotechnology firm that develops and commercializes progressive therapies for sufferers with critical and life-threatening uncommon genetic illnesses. The corporate’s portfolio consists of seven commercialized merchandise and a number of scientific and pre-clinical product candidates. For extra info, please go to www.biomarin.com. Data on such web site isn’t integrated by reference into this press launch.

Ahead-Wanting Statements

This press launch comprises forward-looking statements concerning the enterprise prospects of BioMarin Pharmaceutical Inc. (BioMarin), together with, with out limitation, statements about: BioMarin’s Voxzogo improvement program usually, the continued approval for this indication could also be contingent upon the verification and outline of scientific profit in confirmatory research and BioMarin’s intention to make use of the continuing open-label extension research in comparison with out there pure historical past to meet this post-marketing requirement, the expectation that Voxzogo shall be out there in the US by mid- to late-December and BioMarin’s intention to start the promotion of Voxzogo instantly and advertising authorization opinions in course of in Japan, Brazil, and Australia with potential approvals in these nations in 2022. These forward-looking statements are predictions and contain dangers and uncertainties such that precise outcomes could differ materially from these statements. These dangers and uncertainties embrace, amongst others: ultimate evaluation ongoing scientific trials of Voxzogo; our capability to efficiently manufacture Voxzogo for the scientific trials and commercially, if permitted; the content material and timing of choices by the U.S. Meals and Drug Administration, the European Fee and different regulatory authorities regarding Voxzogo; and people different dangers and uncertainties detailed occasionally below the caption “Threat Elements” and elsewhere within the BioMarin’s Securities and Change Fee (SEC) filings, together with, with out limitation, BioMarin’s Quarterly Report on Kind 10-Q for the quarter ended September 30, 2021, and future SEC filings and experiences by BioMarin. BioMarin undertakes no obligation or obligation to replace any forward-looking statements contained on this press launch because of new info, future occasions or modifications in its expectations.

BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.