Antisense Therapeutics (ASX:ANP) has announced that it has received feedback on the 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 company said PDCO provided feedback outlining additional information requirements for the company’s planned Phase 2b clinical trial in non-ambulant DMD boys. It said "overall" the feedback is "in line with" its expectations.
The company said it anticipates finalising the trial design with PDCO later in the third quarter of the calendar year 2021 ahead of submitting the clinical trial application shortly thereafter.
Approval of the PIP that is currently in development 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 potential ambulant DMD patient studies).
Nuket Desem, director of clinical and regulatory affairs at Antisense Therapeutics, said, “The EMA’s PDCO feedback via the PIP Summary Report was constructive. We have appreciated the collaborative approach of the EMA to assist us in refining the study design for the proposed Phase IIb clinical trial in Europe. It’s another important step towards bringing this new therapy to patients in the EU.”
The company said it is continuing to progress its interactions with the US FDA and has recently submitted a FastTrack Designation Request.
While the Company’s Phase IIb clinical trial protocol was initially developed to meet EMA’s expectations, the recent feedback from the FDA has provided the Company the opportunity to streamline the regulatory processes in Europe and the US and to the extent possible harmonize the Company’s overall global clinical development plans.