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EMA Approves Strensiq For Rare Bone Disorder: Positive CHMP Opinion For Enzyme Replacement Therapy.pdf

chmp-summary-positive-opinion-strensiq_en.pdf
Preview of EMA Approves Strensiq for Rare Bone Disorder: Positive CHMP Opinion for Enzyme Replacement Therapy
🔗 Source: ema.europa.eu
📊 Size: 69 KB
👤 Author: CHMP
⬇️ Downloads: 25

Summary

On June 25, 2015, the Committee for Medicinal Products for Human Use (CHMP) recommended granting a marketing authorization for Strensiq, an orphan medicinal product intended for treating pediatric-onset hypophosphatasia. Alexion Europe SAS is the applicant.

Product Information:
- Active Substance: Asfotase alfa (a human recombinant tissue-nonspecific alkaline phosphatase-Fc-deca-aspartate fusion protein)
- Formulations: 40 mg/ml and 100 mg/ml injection solutions
- Indication: Long-term enzyme replacement therapy for patients with pediatric-onset hypophosphatasia to treat bone manifestations of the disease.

Benefits:
- Improves skeletal structure evident through X-ray, histological bone biopsy, and catch-up height gain in some patients.

Side Effects:
- Most common: Injection site reactions and injection-associated adverse reactions, mostly non-serious and mild to moderate.

Prescription Guidelines:
- Initiation of treatment should be by physicians experienced in managing metabolic or bone disorders.
- Detailed recommendations will be provided in the Summary of Product Characteristics (SmPC) published in the European public assessment report (EPAR).

Authorization Status:
- Granted under exceptional circumstances due to rarity of the condition, limited scientific knowledge, and ethical considerations in data collection.

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Description

The European Medicines Agency's Committee for Medicinal Products recommended granting initial authorisation for Strensiq, an orphan drug treating paediatric-onset hypophosphatasia, manufactured by Alexion Europe SAS. Available in 40 mg/ml and 100 mg/ml solutions.

Technical Information

  • File Format: PDF
  • File Size: 69 KB
  • Pages: 1
  • Language: EN
  • Author: CHMP
  • Total Downloads: 25
  • Last Updated: 4 weeks ago

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