BioMarin is facing competition from Ascendis Pharma’s entry into the achondroplasia field last month and BridgeBio’s entry. To make matters worse, the company experienced a blow after halting two studies of Voxzogo in other growth-related disorders following several incidences of hip injuries in other Voxzogo trials.
Voxzogo by BioMarin has hits that are never-ending. BioMarin is discontinuing dosing and enrollment in trials for Turner syndrome, SHOX deficiency, and aggrecan (ACAN) deficiency, and now that it has an achondroplasia competitor: the FDA approved Ascendis Pharma’s Yuviwel a month ago.
In a Monday SEC filing, BioMarin cited multiple incidences of capital femur epiphysis slipped (SCFE), which occurred in 2 ongoing investigator-sponsored trials of the drug. SCFE is a complication that arises when the rounded head of the femur dislocates at the neck of the thigh bone at the growth plate, bringing about pain, stiffness, and difficulty walking.
In the conditions studied in the investigator-sponsored trials, BioMarin said in the filing, no SCFE events were observed in the Phase 2 trials of Voxzogo in BioMarin, nor in the more than 5,000 children and infants who have undergone Voxzogo in the treatment of achondroplasia, the genetic condition underlying the most common form of dwarfism.
In November 2021, the FDA granted approval to Voxzogo as the first drug to enhance the growth of children with achondroplasia. It is also working on the drug to treat a variety of other growth-related disorders, such as in phase 2 trials of short stature with Turner syndrome and a basket study of Voxzogo in Turner syndrome, SHOX deficiency, and Noonan syndrome (ACAN deficiency).
BioMarin indicated in the SEC filing that two additional Phase 2 trials of Voxzogo will go on as scheduled, one in hypochondroplasia and Noonan syndrome, and individuals with idiopathic short stature (ISS) without acyanotic foraminous dwarfism (ACAN-deficiency). The biotech added to ISS without ACAN-deficiency is about 95% of children enrolled to ISS trial.
Although Voxzogo has been the first market player in achondroplasia, competition has been threatening the company. In the third-quarter earnings call last year, BioMarin had reversed a prior objective of reaching 4 billion in revenue by 2027, a move that was largely informed by emerging competition, as then-CEO Alexander Hardy at the time.
Voxzogo must be administered daily as opposed to the Yuviwel of Ascendis, which is administered weekly, which may give Voxzogo the convenience edge over its competitors.
BridgeBio is also threatening to compete with both companies with infigratinib in a Phase 3 trial last month, which, in a theatrical trial, outperformed even the most optimistic predictions both in efficacy and safety, according to Truist Securities. BridgeBio anticipates submitting a new drug application to the FDA in the second half of the year.
Reason for Suspension
The temporary halt in the Voxzogo studies allows BioMarin to review safety data and assess the ongoing trial protocols. BioMarin emphasized that patient well-being remains its top priority.
Impact on BioMarin’s Clinical Programs
The pause affects multiple sites involved in Voxzogo trials, potentially delaying study timelines. BioMarin is working closely with investigators and regulatory authorities to ensure a careful and transparent process.
Voxzogo Overview
Voxzogo is designed to address rare genetic growth disorders. BioMarin has been investigating its efficacy and safety in multiple age groups to provide meaningful treatment options for patients in need.
The company behind Voxzogo has announced a temporary suspension of dosing and enrollment in two ongoing clinical studies. This decision emphasizes a commitment to patient safety and regulatory compliance during the trials.
Reason for Suspension
The pause allows the research team to review safety data and assess ongoing study protocols. Patient well-being remains the primary concern, ensuring that the trials proceed responsibly.
Impact on Clinical Programs
The suspension affects several trial sites and may cause delays in study timelines. Investigators are working closely with regulatory authorities to maintain careful oversight and transparency.
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