Branaplam, formerly known as LMI070, an oral treatment under investigation for SMA, is currently in a Phase 1/2 clinical trial (safety and efficacy) in untreated SMA Type 1 patients.
Novartis is pleased to announce a couple of developments:
- The expansion in the number of countries and sites for the second part of the trial. Specifically, in sites located in 3 new countries by the end of June 2018. Other countries and sites are also planned and should follow soon. Physicians in other European countries have also reached out to Novartis to participate.
- Reaching an important milestone: On 23rd March, Novartis received the written positive opinion from the European Commission confirming Orphan Drug Designation for branaplam. This follows the Orphan Drug Designation from the US FDA, which was granted on 25th January. The granting of orphan status in the US and EU is intended to support development of medicines for the treatment of rare diseases (less than 5 in 10,000 in the EU). It provides companies certain benefits to encourage the continued development of medicines, like branaplam, that treat small numbers of patients with severe diseases.
Novartis continues to be very active in broader scientific research for SMA. The company introduced a potential biomarker to assess disease severity at our International Scientific Conference in Krakow, Poland. As communicated at the congress, this biomarker seems to be elevated in Type 1 SMA patients when compared to healthy children. Investigations are ongoing.
Novartis continues to work to share information and to talk regularly with the SMA community. The company recently launched its “Novartis Commitment to Patients and Caregivers”. Co-created with over 40 patient organisations, this is a powerful statement that the company commits to embed patient perspectives in its daily work and champion the patient voice in internal discussions and decision making.