BioMarin Presents Real-World Evidence Further Supporting Safety and Efficacy of VOXZOGO ® (vosoritide) in Children with Achondroplasia at the European Society for Paediatric Endocrinology (ESPE) Meeti

SAN RAFAEL, Calif., Nov. 16, 2024 /PRNewswire/ — BioMarin Pharmaceutical (TADAWUL:) Inc. (Nasdaq: BMRN) at the moment introduced optimistic and constant outcomes from a number of real-world proof research of VOXZOGO^® (vosoritide) in youngsters with achondroplasia. These outcomes, in addition to information from the investigational analysis program for VOXZOGO in hypochondroplasia, had been introduced on the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Assembly in Liverpool, England, Nov. 16-18, 2024.

“The intensive medical information supporting VOXZOGO in youngsters with achondroplasia are well-known, and at ESPE, we shared additional leads to youngsters who drastically benefited from the medication in a real-world setting,” mentioned Klaus Mohnike, M.D., Ph.D., pediatric endocrinologist at Kids’s Hospital, Otto-von-Guericke-College in Magdeburg, Germany. “Moreover, the breadth of medical and real-world proof underscores why we proceed to advocate for therapy with VOXZOGO as early as potential.”

Actual-world longitudinal information from the achondroplasia module of the European CrescNet registry involving 452 youngsters at 30 facilities throughout eight nations confirmed outcomes of therapy with VOXZOGO had been according to beforehand reported medical trials. The median age at time of enrollment was 6.12 years. Amongst 143 contributors handled with VOXZOGO for 12 months, the typical top improve was 6.36 centimeters (cm), with a top Z-score enchancment of 0.7 in comparison with an achondroplasia reference inhabitants. For 73 contributors handled for twenty-four months, the typical top improve was 11.86 cm, with a top Z-score enchancment of 1.15 in comparison with the identical reference inhabitants.

As well as, in a real-world examine in France that included 62 youngsters, outcomes from 17 youngsters above the age of 5 (for whom information is on the market for 18 months after preliminary therapy) confirmed continued efficacy of VOXZOGO. The kids who obtained VOXZOGO skilled an 8.76 cm improve in top, on common, and skilled a imply 0.56 enchancment in Z-score in comparison with an untreated pure historical past inhabitants with achondroplasia and a 0.44 enchancment in Z-score in comparison with a basic U.S. inhabitants, indicating progress in top in comparison with the untreated inhabitants. The typical annualized progress velocity was 5.85 cm/yr, representing a considerable enchancment in growth-related improvement over time. There have been no discontinuations, and longer-term security and effectiveness will proceed to be monitored.

“These real-world information additional reinforce the worth of VOXZOGO as the primary and solely authorized therapy for youngsters, together with infants, with achondroplasia,” mentioned Greg Friberg, M.D., government vp and chief analysis & improvement officer at BioMarin. “Now we have now collected greater than 6,000 patient-years of security information by way of our industry-leading CANOPY medical program, offering the scientific foundation for the speedy improvement of VOXZOGO in new indications equivalent to hypochondroplasia, the place we hope to copy our success in achondroplasia.”

Key shows for VOXZOGO in achondroplasia and hypochondroplasia at ESPE, together with two oral shows and 4 poster shows, are listed beneath, with all occasions in Greenwich Imply Time (GMT):

Oral Shows

Section 2 Trial of Vosoritide Use in Sufferers with Hypochondroplasia: Pharmacokinetic/Pharmacodynamic Evaluation from 12 Month Information
Summary #FC5.5
Saturday, Nov. 16 at 3:35 p.m.

Enlargement of the CrescNet Registry Achondroplasia Module: Actual-World Demographic Information and Outcomes After As much as 2 Years of Vosoritide Remedy
Summary #FC2.6
Saturday, Nov. 16 at 3:45 p.m.

Poster Shows

Actual-World Effectiveness of Vosoritide in Kids with Achondroplasia: Outcomes from 18 Months Observe-Up in France
Summary #P2-56

Affected person-Centered Information Assortment Supplies Complete Insights into Healthcare Useful resource Use in Achondroplasia: Information From the Pilot Section of the VIrtual STudy in Achondroplasia (VISTA)
Summary #P1-118

Design and Aims of Research 111-902: a Multicenter, Potential and Retrospective Observational Research of Kids with Hypochondroplasia
Summary #P3-164

Comparability of the Diagnostic Yield of Entire Exome Sequencing (WES) and Focused Panel Sequencing for Kids with Idiopathic Quick Stature (ISS)
Summary #P1-61

Concerning the CANOPY Medical Program

The CANOPY medical program was designed to guage the potential of VOXZOGO (vosoritide) in youngsters with numerous genetic skeletal circumstances, together with achondroplasia, hypochondroplasia, Noonan syndrome, SHOX deficiency, Turner syndrome and idiopathic quick stature, with the objective of addressing the unmet wants of and increasing therapy choices for youngsters and households impacted by these circumstances.

Research underway as a part of the CANOPY program past achondroplasia embody:

CANOPY HCH-OS, a multinational observational examine in youngsters with hypochondroplasia.
CANOPY HCH-3, a Section 3 randomized, placebo-controlled, double-blind multicenter examine in youngsters with hypochondroplasia.
CANOPY ISS-OS, a multinational observational examine in youngsters with idiopathic quick stature.
CANOPY ISS-2, a Section 2 randomized, managed, multicenter examine in youngsters with idiopathic quick stature.
CANOPY NS, TS, SHOX-D-2, a Section 2 examine in a number of genetic skeletal circumstances together with Noonan syndrome, Turner syndrome and SHOX deficiency.

About VOXZOGO

In youngsters with achondroplasia, endochondral bone progress, a necessary course of by which bone tissue is created, is negatively regulated attributable to a acquire of operate mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a optimistic regulator of the signaling pathway downstream of FGFR3 to advertise endochondral bone progress.

VOXZOGO is authorized within the U.S., Japan and Australia to extend linear progress in youngsters of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated within the EU for the therapy of achondroplasia in youngsters 4 months of age and older whose epiphyses are usually not closed, as confirmed by acceptable genetic testing. Within the U.S., this indication is authorized underneath accelerated approval primarily based on an enchancment in annualized progress velocity. Continued approval could also be contingent upon verification and outline of medical profit in confirmatory trial(s). To satisfy this post-marketing requirement, BioMarin intends to make use of the continuing open-label extension research in comparison with accessible pure historical past.

Up to now, roughly 3,800 infants and youngsters with achondroplasia world wide have obtained VOXZOGO.

Affected person Help Accessing VOXZOGO

To succeed in a BioMarin RareConnections^® Case Supervisor, please name, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected]. For extra details about VOXZOGO, please go to www.voxzogo.com. For added data concerning this product, please contact BioMarin Medical (TASE:) Info at [email protected].

About Achondroplasia

Achondroplasia is a uncommon genetic skeletal situation brought on by a variation within the FGFR3 gene. It’s characterised by disproportionate quick stature and a doubtlessly excessive burden of problems associated to impaired endochondral bone progress.

Roughly 80% of kids with achondroplasia are born to folks of common stature on account of a spontaneous variation within the FGFR3 gene. The worldwide incidence of achondroplasia is round one in 25,000 reside births.

VOXZOGO U.S. Essential Security Info

What’s VOXZOGO used for?

VOXZOGO is a prescription medication used to extend linear progress in youngsters with achondroplasia and open progress plates (epiphyses).
VOXZOGO is authorized underneath accelerated approval primarily based on an enchancment in annualized progress velocity. Continued approval could also be contingent upon verification and outline of medical profit in confirmatory trials.

What’s crucial security details about VOXZOGO?

VOXZOGO might trigger severe unwanted side effects together with a brief lower in blood strain in some sufferers. To cut back the chance 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 commonest unwanted side effects of VOXZOGO?

The commonest unwanted side effects of VOXZOGO embody injection web site reactions (together with redness, itching, swelling, bruising, rash, hives, and injection web site ache), excessive ranges of blood alkaline phosphatase proven in blood checks, vomiting, joint ache, decreased blood strain, and stomachache. These are usually not all of the potential unwanted side effects of VOXZOGO. Ask your healthcare supplier for medical recommendation about unwanted side effects, and about any unwanted side effects that hassle the affected person or that don’t go away.

How is VOXZOGO taken?

VOXZOGO is taken day by day as an injection given underneath 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 will have been proven the best manner by your healthcare supplier. VOXZOGO is equipped with Directions for Use that describe the steps for getting ready, injecting, and disposing VOXZOGO. Caregivers ought to overview the Directions for Use for steerage and any time they obtain a refill of VOXZOGO in case any adjustments 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 subsequent day by day dose as regular.
The dose of VOXZOGO is predicated on physique weight. Your healthcare supplier will regulate the dose primarily based on adjustments 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 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 (WA:) your physician about all the 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 medication (anti-hypertensive medication).
- If the affected person has kidney issues or renal impairment.
- If the affected person is pregnant or plans to develop into pregnant. It isn’t identified if VOXZOGO will hurt the unborn child.
- If the affected person is breastfeeding or plans to breastfeed. It isn’t identified if VOXZOGO passes into breast milk.
Inform your physician about all the 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 side effects to BioMarin at 1-866-906-6100. You’re inspired to report detrimental unwanted side effects of pharmaceuticals to the FDA. Go to www.fda.gov/medwatch, or name 1-800-FDA-1088.

Please see extra security data within the full Prescribing Info and Affected person Info.

About BioMarin

BioMarin is a worldwide biotechnology firm devoted to translating the promise of genetic discovery into medicines that make a profound influence on the life of every affected person. The San Rafael, California-based firm, based in 1997, has a confirmed observe report of innovation with eight industrial therapies and a powerful medical and preclinical pipeline. Utilizing a particular method to drug discovery and improvement, BioMarin pursues remedies that supply new potentialities for sufferers and households world wide navigating uncommon or difficult-to-treat genetic circumstances. To study extra, please go to www.biomarin.com.

Ahead-Wanting Statements

This press launch incorporates forward-looking statements in regards to the enterprise prospects of BioMarin Pharmaceutical (NASDAQ:) Inc. (BioMarin), together with with out limitation, statements about: information introduced at European Society for Paediatric Endocrinology (ESPE) 2024, together with the oral and poster shows; VOXZOGO’s efficacy, security and influence on youngsters with achondroplasia, together with the potential advantages of early therapy with VOXZOGO; the potential advantages of VOXZOGO for youngsters with hypochondroplasia; the event of BioMarin’s VOXZOGO program typically, together with BioMarin’s potential to quickly develop of VOXZOGO in new indications equivalent to hypochondroplasia; and BioMarin’s CANOPY medical program, together with BioMarin’s plans and expectations for medical trials for numerous genetic skeletal circumstances, together with achondroplasia, hypochondroplasia, Noonan syndrome, SHOX deficiency, Turner syndrome and idiopathic quick stature. These forward-looking statements are predictions and contain dangers and uncertainties such that precise outcomes might differ materially from these statements. These dangers and uncertainties embody, amongst others: outcomes and timing of present and deliberate pre-clinical research and medical trials of VOXZOGO; any potential opposed occasions noticed within the persevering with monitoring of the sufferers within the medical trials; the content material and timing of selections by the Meals and Drug Administration, the European Fee and different regulatory authorities; and people elements detailed in BioMarin’s filings with the Securities and Trade Fee, together with, with out limitation, the elements contained underneath the caption “Danger Elements” in BioMarin’s Quarterly Report on Kind 10-Q for the quarter ended September 30, 2024, as such elements could also be up to date by any subsequent experiences. Stockholders are urged to not place undue reliance on forward-looking statements, which communicate solely as of the date hereof. BioMarin is underneath no obligation, and expressly disclaims any obligation to replace or alter any forward-looking assertion, whether or not on account of new data, future occasions or in any other case.