Achondroplasia

New Data to be Presented for BioMarin's VOXZOGO® (vosoritide) in Children with Hypochondroplasia and Achondroplasia at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

Retrieved on: 
Tuesday, March 12, 2024
Quality of life, ACMG, SAN, Medical genetics, AGV, American College, Observation, Achondroplasia, Hypochondroplasia, Medicine

SAN RAFAEL, Calif., March 12, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024. Researchers will also present data from Phase 2 and Phase 3 studies of the medicine in children with achondroplasia, including results that demonstrate VOXZOGO's positive impact on quality of life. 

Key Points: 
  • Andrew Dauber, M.D., will present positive results from his investigator-sponsored Phase 2 study of VOXZOGO in children with hypochondroplasia.
  • The annualized growth velocity (AGV) increased from 5.12 cm/year during the observation period to 6.93 cm/year during the treatment period (mean difference: 1.81 cm/year, p
  • In late 2023, BioMarin launched the pivotal clinical trial program studying the safety and efficacy of VOXZOGO in children with hypochondroplasia.
  • "Our studies showed that increasing height with long-term administration of VOXZOGO can result in meaningful improvements in quality-of-life measures for children with achondroplasia."

Ascendis Pharma Reports Fourth Quarter and Full Year 2023 Results

Retrieved on: 
Wednesday, February 7, 2024
Investigational New Drug, Oncology, Endocrine gland, Research, GHD, Patient, New Drug Application, CNP, FDA, Achondroplasia, PDUFA, Turner syndrome, Webcast, Pharmaceutical industry

Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.

Key Points: 
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia.
  • Total revenue for the fourth quarter of 2023 was €137.7 million compared to €22.9 million during the same period for 2022.
  • Ascendis Pharma will also host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss 2023 financial results.

BridgeBio Pharma and Kyowa Kirin Announce Partnership with an Upfront Payment of $100 Million for an Exclusive License on Infigratinib in Skeletal Dysplasias in Japan

Retrieved on: 
Wednesday, February 7, 2024
Partnership, PALO, License, USD, Osteochondrodysplasia, Child, Safety, MBA, FGFR3, Achondroplasia, Therapy, Kyowa Kirin, Hypochondroplasia, Pharmaceutical industry

In exchange, BridgeBio will receive an upfront payment of USD 100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.

Key Points: 
  • In exchange, BridgeBio will receive an upfront payment of USD 100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.
  • Infigratinib is an oral small molecule designed to inhibit FGFR3 and thus target FGFR3-driven skeletal dysplasias at their source, including achondroplasia and hypochondroplasia.
  • “By partnering with Kyowa Kirin, we hope to significantly accelerate the development of infigratinib to potentially provide options for children with achondroplasia, hypochondroplasia, and eventually skeletal dysplasias in Japan.
  • Based on the results from the latest clinical trials, we believe BridgeBio’s infigratinib has high potential for treating achondroplasia.

Er-Kim Announces Exclusive Distribution Agreement with Ascendis Pharma A/S to Commercialize 3 Endocrinology Disease Treatments Across Central & Eastern Europe and Turkey

Retrieved on: 
Monday, January 8, 2024
Achondroplasia, CFO, ACH, European, Osteochondrodysplasia, PTH, Endocrinology, Patient, Phase II, Pharmaceutical industry

BUCHAREST, Romania, Jan. 09, 2024 (GLOBE NEWSWIRE) -- Er-Kim , an international pharmaceutical company specializing in the commercialization of novel therapies, today announced it has entered into an exclusive distribution agreement with Danish Company Ascendis Pharma A/S (Nasdaq: ASND) for its endocrinology portfolio.

Key Points: 
  • BUCHAREST, Romania, Jan. 09, 2024 (GLOBE NEWSWIRE) -- Er-Kim , an international pharmaceutical company specializing in the commercialization of novel therapies, today announced it has entered into an exclusive distribution agreement with Danish Company Ascendis Pharma A/S (Nasdaq: ASND) for its endocrinology portfolio.
  • Under the agreement, Er-Kim is appointed as the exclusive Ascendis A/S endocrinology distributor in Albania, Bosnia and Herzegovina, Bulgaria, Croatia, Cyprus, Czechia, Estonia, Greece, Hungary, Kosovo, Latvia, Lithuania, Malta, Montenegro, North Macedonia, Poland, Romania, Serbia, Slovakia, Slovenia, and Türkiye.
  • He added, “Leveraging our exceptional expertise in rare diseases and our preeminent status in endocrinology throughout the region, we eagerly anticipate collaborating with Ascendis and the healthcare community.
  • “We are therefore very pleased to partner with Er-Kim, a company that shares this commitment, to broaden and accelerate access to our innovative Endocrinology Rare Disease treatment options across Central and Eastern Europe and Turkey.”

Ascendis Pharma Introduces Vision 2030

Retrieved on: 
Sunday, January 7, 2024
Growth hormone, Vision 2030, Patient, Endocrine gland, Hypoparathyroidism, Conference, Achondroplasia, Turner syndrome, FDA, GHD, PDUFA, Globalization, Investigational New Drug, New Drug Application, Transgender hormone therapy, J. P. Morgan, Ophthalmology, CNP, HNSCC, Growth, CTA, Pharmaceutical industry

COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference.

Key Points: 
  • COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030.
  • Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com .

Specialised Therapeutics signs exclusive agreement with Ascendis Pharma A/S for distribution and commercialisation of three endocrinology therapies in Australia and select South-East Asia countries

Retrieved on: 
Sunday, January 7, 2024
Growth hormone, Hypoparathyroidism, Quality of life, Partnership, Achondroplasia, Vitamin D, ACH, European, Disease, Osteochondrodysplasia, Therapy, PTH, Endocrinology, CNP, Patient, Phase II, Pharmaceutical industry

Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFATM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATHTM (palopegteriparatide) and investigational achondroplasia therapy TransCon™ CNP (navepegritide).

Key Points: 
  • Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFATM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATHTM (palopegteriparatide) and investigational achondroplasia therapy TransCon™ CNP (navepegritide).
  • The agreement spans ST's key regions of Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam.
  • Announcing the partnership, ST Chief Executive Officer Carlo Montagner said this agreement was an important company milestone, signalling ST's expansion into both endocrinology and paediatric medicine.
  • "We are delighted to partner with Specialised Therapeutics to broaden the reach of our endocrinology rare disease portfolio, contributing to our shared goal of making a meaningful difference for patients facing unmet medical needs," she said.

Significant Health and Quality of Life Improvements Achieved in Children with Achondroplasia Treated for One Year with TransCon™ CNP (Navepegritide) at 100 µg/kg/week

Retrieved on: 
Wednesday, December 20, 2023
Achondroplasia, CNP, Growth, Patient, OLE

In the trial, all 57 patients have now completed one year of treatment with TransCon CNP (navepegritide) at 100 µg/kg/week, the dose agreed with regulatory agencies for the active arm in the pivotal ApproaCH Trial.

Key Points: 
  • In the trial, all 57 patients have now completed one year of treatment with TransCon CNP (navepegritide) at 100 µg/kg/week, the dose agreed with regulatory agencies for the active arm in the pivotal ApproaCH Trial.
  • “These data support our aspirations for TransCon CNP, the first investigational product to go beyond height by improving the signs and symptoms of achondroplasia.
  • We believe that the observed treatment benefits are likely contributing to the continued strong patient retention in our trials,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma.
  • Further details of the results will be shared early next year.

BridgeBio Announces First Child Dosed in PROPEL 3, its Phase 3 Clinical Trial for Infigratinib in Children with Achondroplasia

Retrieved on: 
Wednesday, December 13, 2023
ENDO, Hypochondroplasia, Achondroplasia, AHV, Research, FDA, PALO, Murdoch, Cancer, PROPEL, Growth, Safety, Child, Pharmaceutical industry

PALO ALTO, Calif., Dec. 13, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first child has been dosed in PROPEL 3, a Phase 3 clinical trial studying the efficacy and safety of infigratinib in children with achondroplasia. Both the U.S. FDA and the EU EMA indicated the trial design for PROPEL 3 would be acceptable as a registrational study to support a marketing application for the treatment of children with achondroplasia.

Key Points: 
  • “The Phase 2 data for infigratinib has been very promising and suggests potential to increase growth, improve functionality and reduce the associated medical complications in children with achondroplasia.
  • PROPEL 3 is a global, one-year, 2:1 randomized, double-blinded placebo-controlled clinical trial, which will evaluate the efficacy and safety of infigratinib in children with achondroplasia aged 3 to
  • Information about PROPEL (NCT04035811), BridgeBio’s observational lead-in study in achondroplasia for PROPEL 3 and other studies, can be found here on clinicaltrials.gov.
  • BridgeBio is committed to exploring the potential of infigratinib on wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, which hold significant unmet needs for families.

Ascendis Pharma Reports Third Quarter 2023 Financial Results

Retrieved on: 
Tuesday, November 7, 2023
FDA, Research, IND, CHMP, European Commission, Oncology, SG, Pathway, European Directive on Traditional Herbal Medicinal Products, GHD, Southern Transcon, Carcinoma, R, Achondroplasia, SDS, OLE, Enlightenment, European Medicines Agency, Webcast, CNP, Keep Reachin Up, All Our Relations: Finding the Path Forward, Endocrine gland, NDA, Safety, Growth hormone, Patient, Drug discovery, Pharmaceutical industry, Ophthalmology

Topline results from Phase 3 foresiGHt Trial in adult growth hormone deficiency expected in the fourth quarter of 2023, potentially opening a new label expansion opportunity.

Key Points: 
  • Topline results from Phase 3 foresiGHt Trial in adult growth hormone deficiency expected in the fourth quarter of 2023, potentially opening a new label expansion opportunity.
  • As of September 30, 2023, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €455.4 million compared to €742.9 million as of December 31, 2022.
  • Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its third quarter 2023 financial results.
  • The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com .

BioMarin Announces Retirement of Jean-Jacques Bienaimé; Appoints Alexander Hardy President and Chief Executive Officer

Retrieved on: 
Wednesday, November 1, 2023
Science, DSM-IV codes, Pathogenesis, Haemophilia, Ophthalmology, Organization, Research, Achondroplasia, Enzyme replacement therapy, Growth, Novartis, Neuroscience, Roche, GSK, JJ, Patient, BioMarin Pharmaceutical, Management, Pharmaceutical industry, Shareholder, Genentech

SAN RAFAEL, Calif., Nov. 1, 2023  /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, announced today that Alexander Hardy has been named president and chief executive officer, succeeding Jean-Jacques Bienaimé, who will retire as chairman and CEO effective December 1, 2023. Mr. Hardy, currently the CEO of Genentech, will also serve as a director on BioMarin's board of directors. Richard A. Meier, BioMarin's lead independent director, will assume the role of chair of the board of directors.

Key Points: 
  • Mr. Hardy, currently the CEO of Genentech, will also serve as a director on BioMarin's board of directors.
  • Richard A. Meier, BioMarin's lead independent director, will assume the role of chair of the board of directors.
  • Mr. Hardy, 55, brings more than 30 years of experience in the global healthcare and biotechnology industries, most recently serving as CEO of Genentech, a member of the Roche Group.
  • Thanks to JJ, BioMarin is ideally positioned for its next chapter with Alexander at the helm."