Achondroplasia

Tyra Biosciences Announces Appointments of Susan Moran, M.D., M.S.C.E. and S. Michael Rothenberg, M.D., Ph.D. to its Board of Directors

Retrieved on: 
Tuesday, May 7, 2024
Stanford University, University, FGFR, Disorder, Fibroblast growth factor receptor, Hospital, NDA, QED, Achondroplasia, Hematology, Bile duct, Drug, Research and development, MAA, Yale University, RET, Growth, Duke University, Biotechnology, Therapy, Faculty, Osteochondrodysplasia, Harvard Medical School, Multiple sclerosis, Biology, Drug development, Drug discovery, Liver cancer, Bristol Myers Squibb, Massachusetts General Hospital, Puma Biotechnology, Pharmaceutical industry

and S. Michael Rothenberg, M.D., Ph.D. as independent directors, and the resignation of Isan Chen, M.D.

Key Points: 
  • and S. Michael Rothenberg, M.D., Ph.D. as independent directors, and the resignation of Isan Chen, M.D.
  • "Susan and Michael's impressive careers will provide TYRA with valuable guidance as we advance our potential next-generation precision candidates toward people in need.
  • In connection with these appointments, Dr. Chen, who has served as a director since June 2020, has resigned from the TYRA Board.
  • "On behalf of the Board, I want to thank Isan for his many contributions to TYRA.

Ascendis Pharma Reports First Quarter 2024 Financial Results

Retrieved on: 
Thursday, May 2, 2024
FDA, Endocrine gland, Hypoparathyroidism, Element, GHD, Achondroplasia, Commercial, New Drug Application, Biologics license application, Development, Prescription, ASCO, Turner syndrome, PDUFA, Patient, Society, Melanoma, Vision, Inborn errors of metabolism, Pharmaceutical industry

If approved, U.S. launch planned in Q3

Key Points: 
  • If approved, U.S. launch planned in Q3
    — SKYTROFA® Q1 revenue more than doubled year-over-year to €65 million; Q1 operating expenses fell by 20% year-over-year to €137 million
    COPENHAGEN, Denmark, May 02, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Plan to submit a supplemental Biologics License Application to FDA for adult growth hormone deficiency (GHD), in the third quarter of 2024.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • In the U.S., Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.

First clinical trial of vosoritide for children with hypochondroplasia shows increased growth

Retrieved on: 
Friday, April 12, 2024
BioMarin Pharmaceutical, Achondroplasia, FGFR3, Noonan syndrome, Female, Costello syndrome, SDS, Observation, Patient, Manuscript, NPR2, Clinical trial, Chart, Growth

“This is the first medicine that has been developed to specifically target the pathway involved in hypochondroplasia,” says Andrew Dauber, M.D.

Key Points: 
  • “This is the first medicine that has been developed to specifically target the pathway involved in hypochondroplasia,” says Andrew Dauber, M.D.
  • “These findings will help inform future studies of vosoritide for addressing growth disorders.”
    This clinical trial funded by BioMarin is the first-of-its-kind to treat children with genetic short stature who do not have achondroplasia.
  • Hypochondroplasia specific height SDS increased by 0.38 SD, according to the trial published in eClinicalMedicine .
  • The study findings also showed standing height SDS increased by 0.37 SD during the year of treatment or 0.41 SD using hypochondroplasia specific growth charts.

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.