Modification des conditions de l’inscription et réévaluation à la demande de la CT. Avis favorable au remboursement dans « le traitement de l’achondroplasie chez les patients âgés de 4 mois à moins de...

BioMarin Pharmaceutical's flagship therapy Voxzogo is on track to surpass $1.0 billion in revenue for 2026, driven by a 20% surge in global patient demand, executives said at the Citi Biopharma Summit. The projected full-year 2026 revenue of $1.0 billion to $1.05 billion highlights sustained growth

BioMarin Pharmaceutical is expanding its rare-disease portfolio as VOXZOGO revenue growth accelerates. This performance led the company to raise its full-year sales guidance for the treatment to a $1 billion midpoint. The results reflect expansion within the firm's metabolic conditions segment, whic

Na jaren wachten en onzekerheid is er eindelijk goed nieuws voor Sem Delvaux (10) uit Houthalen-Helchteren. Het peperdure geneesmiddel Voxzogo dat de jongen moet helpen groeien, wordt straks eindelijk terugbetaald. “Hier durfden we eigenlijk niet meer op te hopen”, zegt een dolgelukkige mama Evelien Geuens.

Reported Tuesday, Sep 15 - 8:35am EDT (TheFly subscription required to read more.)

Click to view the official SEC document.

BioMarin Pharmaceuticalは2026年9月9日、軟骨無形成低身長症（hypochondroplasia）を対象とした第3相CANOPY-HCH-3試験でVOXZOGO（vosoritide）が主要評価項目を達成したと発表した。成長速度や身長など複数指標の結果と安全性、FDA申請の状況を整理する。

A Phase 3 multicenter study has demonstrated that vosoritide, marketed as VOXZOGO, significantly improved growth in children aged 3 to 17 years with hypochondroplasia, a rare genetic skeletal conditio

BioMarin has reported significant improvements in growth measures for VOXZOGO, aimed at treating hypochondroplasia in children, during Phase 3 trials.

Reported Tuesday, Aug 18 - 4:35pm EDT (TheFly subscription required to read more.)

BioMarin Pharmaceutical Inc. reports impressive financial results for Q2 2026, achieving a 20% revenue increase year-over-year and raising full-year guidance.

BioMarin (NASDAQ:BMRN) reported second quarter 2026 total revenues of $990 million, up 20% year-over-year, driven mainly by newly acquired GALAFOLD and POMBILITI + OPFOLDA, VOXZOGO growth, and higher PALYNZIQ demand. Metabolic Conditions revenue rose 25% Y/Y to $695 million.GAAP net income fell to $45 million from $241 million, and Non-GAAP income declined to $236 million from $282 million, reflecting Amicus acquisition-related costs, higher interest expense, and increased commercial and R&D spending. GAAP diluted EPS was $0.23 versus $1.23; Non-GAAP diluted EPS was $1.20 versus $1.44.BioMarin raised full-year 2026 guidance, with total revenues now expected between $3.875–$3.925 billion, VOXZOGO revenues between $1.0–$1.05 billion, and Non-GAAP diluted EPS between $4.90–$5.10. The company identified about $280 million in GAAP cost reductions from the Amicus deal, targets gross leverage below 2.5x by mid-2027, and projects high-margin growth from GALAFOLD and POMBILITI + OPFOLDA.Pipeline updates include positive Phase 3 hypochondroplasia data and an sNDA for VOXZOGO, Phase 1 data for BMN 333, addition of BMN 820, continued development of BMN 351, discontinuation of BMN 401, and an ASO collaboration with n-Lorem for ReNU syndrome.

 > Retouradres Postbus 20350 2500 EJ Den Haag De Voorzitter van de Tweede Kamer der Staten-Generaal Postbus 20018 2500 EA DEN HAAG Datum 16 juli 2026 Betreft inzake voor het zomerreces met de fabrikant van Voxzogo om tafel gaan om te komen tot indiening van het dossier bij het Zorginstituut Geachte voorzitter, Zoals toegezegd in het tweeminutendebat Pallas van 2 juli 2026, informeert het kabinet de Kamer hierbij over de reactie van de leverancier van het geneesmiddel Voxzogo, BioMarin, op de oproep vanuit het ministerie van VWS om het besluit om voor Voxzogo geen vergoedingsdossier in te dien…

Application based on long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span evaluated over long-term follow-up FDA PDUFA target action date of Feb. 28, 2027...

BioMarin Pharmaceutical announces the FDA's acceptance of VOXZOGO® for full approval in children with achondroplasia. Aiming for enhanced skeletal growth and well-being, the full approval is anticipated by early 2027.

Reported Monday, Jul 13 - 9:07am EDT (TheFly subscription required to read more.)

BioMarin (Nasdaq:BMRN) announced that the U.S. FDA has accepted its supplemental New Drug Application (sNDA) seeking full approval of VOXZOGO (vosoritide) for children with achondroplasia, and set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.According to BioMarin, the sNDA is supported by long-term safety and efficacy data from three ongoing studies (111-205, 111-208, 111-302), including adult height and additional clinical outcomes such as body proportionality and arm span. The submission is intended to fulfill the postmarketing requirement for converting VOXZOGO’s 2021 accelerated approval to traditional approval, backed by what the company describes as the longest efficacy and safety data package for a medicine studied in achondroplasia.