Antisense Therapeutics (ASX:ANP) has announced that it has received a draft opinion recommending the agreement with its Paediatric Investigation Plan (PIP) for the development of ATL1102 for Duchenne muscular dystrophy (DMD) from the Paediatric Committee (PDCO) of the European Medicines Agency (EMA).
The draft opinion says, "The Paediatric Development Committee, having assessed the proposed paediatric investigation plan in accordance with Article 17 of Regulation (EC) No 1901/2006 as amended recommends as set out in the appended summary report to agree the paediatric investigation plan in accordance with Article 17(1) of said Regulation."
The company said the measures and timelines of the paediatric investigation plan recommended for agreement are set out in the draft opinion. The draft opinion is currently under review before discussion and adoption by PDCO during their meeting on 15 October, with the company requested to provide feedback including checking the document for any inaccuracies ahead of the meeting.
"The opinion is draft and may not reflect the adopted PDCO opinion," said the company, adding it is not aware of any material issues that would adversely impact PCDO’s adoption of the draft opinion.
"The draft opinion appears entirely consistent with the Company’s plans for the conduct of the Phase 2b trial," it said.
A paediatric investigation plan is a development plan aimed at ensuring that the necessary data is obtained through studies in children. Approval of the PIP is required to support the authorisation of a medicine for children in the European Union (EU). The PIP addresses the entire paediatric development program for ATL1102 in DMD (including future ambulant DMD patient studies).
Antisense said it is looking to ensure that its planned clinical studies including its Phase 2b clinical trial of ATL1102 in non-ambulant DMD boys, will be run in accordance with PDCO expectations for future product approval.
According to Nuket Desem, the director of clinical and regulatory affairs at Antisense Therapeutics, “Receipt of the EMA’s PDCO positive feedback on the PIP is another key step forward in advancing the Company’s plans for the conduct of the potentially approvable Phase IIb study of ATL1102 in DMD in Europe, in line with the Company’s expectations. With the final opinion due in the coming weeks, we are finalising the site evaluations and selections for the conduct of the trial and progressing preparations of the clinical trial applications for submission to the national competent authorities.”