Wednesday, July 2, 2025
Distribution: (800) 510 0384
Washington DC
New York
Toronto
Press ID  
  • Login
The Hudson Weekly
  • Financial
  • Blockchain
  • Technology
  • Entertainment
  • Lifestyle
  • Arts
  • Health
  • Sports
  • Cybersecurity
No Result
View All Result
  • Financial
  • Blockchain
  • Technology
  • Entertainment
  • Lifestyle
  • Arts
  • Health
  • Sports
  • Cybersecurity
No Result
View All Result
The Hudson Weekly
No Result
View All Result

FDA Accepts BioMarin’s New Drug Application for Vosoritide to Treat Children with Achondroplasia

If approved, 1st Therapy in U.S. for the Treatment of Achondroplasia. PDUFA Action Date is Aug. 20, 2021

Emily Manifold by Emily Manifold
November 18, 2020
in Health
A A
FDA Accepts BioMarin’s New Drug Application for Vosoritide to Treat Children with Achondroplasia

© FDA

Share on FacebookShare on Twitter

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. This acceptance by the FDA marks the first marketing application accepted for a treatment for achondroplasia in the United States

The Prescription Drug User Fee Act (PDUFA) action date is Aug. 20, 2021. The FDA has informed the company that they are not currently planning to hold an advisory committee meeting to discuss the application.

HudsonNewsroom

Beyond the Headlines: What’s Driving Clinical Trials in Eastern Europe?

Tips to Buy Cylinder Carts: What You Need to Know

The Science of Healthy Hair: Essential Habits for Everyday Styling

Although the FDA did not identify any filing issues with the NDA, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups. BioMarin believes the highly persuasive outcomes from the one-year randomized, double-blind, placebo-controlled Phase 3 trial, coupled with data from the Phase 2 program with up to 5 years of long-term follow-up that has been compared to robust natural history data on growth, offers a rigorous and reliable method to assess whether vosoritide has a durable impact on the rate of endochondral bone growth that ultimately increases final adult height. This information was included in the marketing application.

While not part of the vosoritide marketing application, in Q4, the Company is also expecting to complete enrollment in a Phase 2 randomized, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia, aged zero to less than 60 months, for a period of 52 weeks. The study will be followed by a subsequent open-label extension trial when all subjects receive active treatment. Children in this study will complete a minimum three-month baseline study to determine their respective baseline growth prior to dosing in the Phase 2 study. Children in this study will have completed a minimum three-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age-matched, average stature children. The company also plans to augment the height Z-score data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.

“We are looking forward to working with the FDA and other regulatory authorities to evaluate the safety and efficacy of the potentially first pharmacological treatment for children with achondroplasia. In addition to the completed clinical studies included in the application, we have included a natural history study to understand the progression of achondroplasia and medical implications,” said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin. “Our extensive development program has been focused on addressing the underlying cause of achondroplasia. We continue to respect the community’s range of views around treatment options. We are grateful to the families who have participated in the clinical trials, the advocacy groups from around the world, and the study investigators, all who have been on this journey with us to deliver a treatment choice and contribute to further understanding achondroplasia medically and scientifically.”

“This is important progress that could lead to the first pharmacological treatment of the underlying cause of achondroplasia. As a treating physician, I feel that it is very important that such treatment options become available to families who may want to consider them for their child,” said John A. Phillips III, M.D., Vanderbilt University Medical Center (David T Karzon Professor of Pediatrics) and investigator for the vosoritide clinical program. “The comprehensive clinical program for vosoritide provides scientific and clinical knowledge that focuses on the safety and efficacy of vosoritide and better understanding of the basic cause of achondroplasia, its natural history and treatment.”

“We are hopeful that our community is one step closer to having access to the first therapeutic choice for children and families affected by achondroplasia,” said Munira Haider, co-founder of Growing Stronger. “We advocate for rigorous scientific and medical research to improve the quality of medical care for little people.”

Growing Stronger’s mission is to improve the quality of medical care for people through supporting research. The organization raises nonprofit donations that are granted to researchers focused on dwarfism.

Regulatory Status in the U.S. and Europe

BioMarin previously announced that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application. Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

Emily Manifold

Emily Manifold

Newsdesk Assistant Editor

More from HW Newsdesk

Beyond the Headlines: What’s Driving Clinical Trials in Eastern Europe?
Health

Beyond the Headlines: What’s Driving Clinical Trials in Eastern Europe?

July 1, 2025
Tips to Buy Cylinder Carts: What You Need to Know
Health

Tips to Buy Cylinder Carts: What You Need to Know

July 1, 2025
The Science of Healthy Hair: Essential Habits for Everyday Styling
Health

The Science of Healthy Hair: Essential Habits for Everyday Styling

June 25, 2025

HW Newsroom

Freddy Will
Entertainment

Revival & Resilience: The Unstoppable Freddy Will

by Dennis Keller
July 1, 2025

In an era where digital music and literary distribution intersect with relentless cyber threats, Grammy-nominated artist and author Freddy Will...

Finding Your Match: Selecting the Ideal Casino Game

Is Luck or Logic Winning More in Poker Now?

July 1, 2025
Understanding Caregiver Insurance: What You Need to Know

Understanding Caregiver Insurance: What You Need to Know

July 1, 2025
Reasons Why You Need a High-Quality Mouse Pad

Reasons Why You Need a High-Quality Mouse Pad

July 1, 2025
Why Ignoring HVAC Maintenance Can Cost You More in the Long Run

Why Ignoring HVAC Maintenance Can Cost You More in the Long Run

July 1, 2025
Tips to Buy Cylinder Carts: What You Need to Know

Tips to Buy Cylinder Carts: What You Need to Know

July 1, 2025
Benefits of Boat and RV Services in Austin

Benefits of Boat and RV Services in Austin

July 1, 2025
Why These Jewish Symbols Still Matter Today

Why These Jewish Symbols Still Matter Today

July 1, 2025
What Jewish Symbols Really Mean: From Ancient Art to Modern Judaica

What Jewish Symbols Really Mean: From Ancient Art to Modern Judaica

July 1, 2025
A Vivid Window Into the Past, A Beacon of Hope for the Future

A Vivid Window Into the Past, A Beacon of Hope for the Future

June 30, 2025
Preventative Botox®: The Strategy for Keeping Skin Smooth Before Wrinkles Set In

Preventative Botox®: The Strategy for Keeping Skin Smooth Before Wrinkles Set In

June 27, 2025
9 Most Popular Gaming Genres

4 Tips for Safe Online Gaming

June 27, 2025
No Result
View All Result

Headlines

The Dirtiest Places in Your Home — And You’re Probably Ignoring Them

Yevhen Kasyanenko on Smart Contracts: How Blockchain Technology Works in Simple Terms

Ghana’s Best Licensed Betting Platforms – Safe & Legal Bookmakers

Revival & Resilience: The Unstoppable Freddy Will

Is Luck or Logic Winning More in Poker Now?

Understanding Caregiver Insurance: What You Need to Know

Trending

Key Benefits of Working With a Custom Application Development
Technology

Key Benefits of Working With a Custom Application Development

by Ryan Offman
July 1, 2025

Last month, I spoke with Sarah, who runs a mid-sized logistics company. She'd been struggling with her...

Beyond the Headlines: What’s Driving Clinical Trials in Eastern Europe?

Beyond the Headlines: What’s Driving Clinical Trials in Eastern Europe?

July 1, 2025
The Dirtiest Places in Your Home — And You’re Probably Ignoring Them

The Dirtiest Places in Your Home — And You’re Probably Ignoring Them

July 1, 2025
Yevhen Kasyanenko on Smart Contracts: How Blockchain Technology Works in Simple Terms

Yevhen Kasyanenko on Smart Contracts: How Blockchain Technology Works in Simple Terms

July 1, 2025
Where Gamblers Go When They Want More: Exploring Unconventional Casino Choices

Ghana’s Best Licensed Betting Platforms – Safe & Legal Bookmakers

July 1, 2025
  • Kia America представляет цены и захватывающие обновления для Sorento 2025 (ICE)

https://madisongraph.com/kia-america-unveils-pricing-and-exciting-updates-for-2025-sorento-ice/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost
  • Kia Recognized as One of TIME Magazine’s “World’s Most Sustainable Companies of 2024”

https://madisongraph.com/kia-recognized-as-one-of-time-magazines-worlds-most-sustainable-companies-of-2024/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost
  • VinFast Auto Establishes Dealer Advisory Board to Enhance Customer Experience

https://madisongraph.com/vinfast-auto-establishes-dealer-advisory-board-to-enhance-customer-experience/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost
  • Medicare Announces Expansion of Coverage for Microprocessor-Controlled Knees for Lower Mobility Users

https://lincolncitizen.com/medicare-announces-expansion-of-coverage-for-microprocessor-controlled-knees-for-lower-mobility-users/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost
  • GA-ASI Successfully Tests PT6 Engine on MQ-9B SkyGuardian Aircraft

https://lincolncitizen.com/ga-asi-successfully-tests-pt6-engine-on-mq-9b-skyguardian-aircraft/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost
  • U.S. Leading Economic Index Declines by 0.2% in June 2024, Coincident Index Rises by 0.3%

https://fairmontpost.com/u-s-leading-economic-index-declines-by-0-2-in-june-2024-coincident-index-rises-by-0-3/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost
  • USTDA Director Travels to Bulgaria and Romania to Promote Clean, Secure Energy

https://belmontstar.com/ustda-director-travels-to-bulgaria-and-romania-to-promote-clean-secure-energy/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost
  • Police Recommend a Hidden Camera Detector Device to Protect Privacy

https://marketsherald.com/police-recommend-a-hidden-camera-detector-device-to-protect-privacy/

#nyc #losangeles #chicago #houston #phoenix #philadelphia #sandiego #dallas #sanfrancisco #seattle #denver #washingtondc #boston #detroit #vancouver #toronto #publicrelations #marketingagency #earnedmedia #editorial #marketing #guestpost #guestposting #sponsored #sponsoredpost

© 2025 The Hudson Weekly. Published by The Ritz Herald. Editions: Markets Herald • Lincoln Citizen • Madison Graph • Belmont Star • Fairmont Post

Address: 1177 6th Avenue, 5th Floor, New York, NY 10036. Removals: pr@hudsonweekly.com. Phone: (718) 313-5252. M-F: 9AM-5PM. Privacy Policy

Welcome Back!

Login to your account below

Forgotten Password?

Retrieve your password

Please enter your username or email address to reset your password.

Log In

Add New Playlist

No Result
View All Result
  • Financial
  • Blockchain
  • Technology
  • Entertainment
  • Lifestyle
  • Arts
  • Health
  • Sports
  • Cybersecurity

© 2025. The Hudson Weekly