Enabling a biotech enter US and EU markets with a rare neurodegenerative disease treatment

An Asia Pacific based biotech was developing a therapeutic treatment for a neurodegenerative disorder. They recognised their drug’s potential to treat a different rare neurodegenerative disorder. With the aim of expanding into new regions, the sponsor sought support from ICON Regulatory Consulting Solutions for submissions to EU and USA regulatory authorities. If successful, their drug would be a first-to-market treatment for a rare disease with an unmet medical need. A phase 2 study was conducted at multiple sites in Asia. The sponsor sought permission to run a similar phase 2 study in the US.

The sponsor wanted to market the new therapy within the shortest feasible time using region-specific regulatory pathways for medicines that serve unmet needs. To do this they needed to register a phase 2 study in the US based on data from study participants in Asia. Orphan Drug Designation (ODD) in the US and EU, and Fast Track Designation (FTD) in the US can accelerate product development and communications with regulatory authorities. This can potentially lead to market exclusivity, lower development costs and faster drug development. However, without regulatory approval the development plan would face significant delays and miss these potential benefits. The biotech was in need of expert regulatory affairs services in this therapeutic area and in EU and US regulations.

ICON Regulatory Consulting Solutions reviewed the available data from the Asian studies and the clinical development plan (CDP). They identified shortcomings which could cause the EU and US regulators to refuse approval within the desired timeframe. Review focus included:

• Dosing rationale
• Suitability of non-clinical pharmacology studies to support clinical development
• Long-term treatment of patients
• Choice and timing of primary endpoints
• Selection of secondary endpoints

In collaboration with colleagues in ICON Drug Development Solutions and Patient Centred Outcomes, ICON revised the CDP to better meet regulatory expectations and anticipate queries. Plans were developed for studies beyond the phase 2 trial and a long-term extension design outlined to provide the necessary data for the regulators. By extending the study, participants could continue to use the treatment after the double-blind period. This would benefit recruitment and retention, giving rare disease patients access to a new treatment. Primary and secondary endpoints were revised based on ICON’s recommendations. Crucially, the revised plan included digital endpoints to demonstrate clinically significant treatment outcomes which would not have been captured otherwise.

ICON supported the client to prepare and submit meeting packages in the US and EU and the subsequent IND submission for a US clinical trial. ICON also gave strategic advice on applying for ODD and FTD grants for the sponsor’s treatment for this neurodegenerative disease indication.

The sponsor’s meetings with EU and US regulatory authorities resulted in broad support for the revised CDP. ICON’s feedback supported revised endpoints with an appropriate number of outcomes to represent trial participants’ expected experiences during successful treatment. Non-clinical support services supported the review of three additional studies. The sponsor received an initial IND without hold comments following successful meetings with EU and US agencies. Significantly, the treatment secured EU and US ODD grants and a FTD grant in the US. These designations can result in potential savings of time and cost to develop the product and bring it to market. Recognising our support, the sponsor said;

“I’m deeply grateful for your dedication and hard work. I believe ICON is the best partner of our drug’s future growth and success.”

The sponsor’s high satisfaction is evident in their ongoing relationship with ICON for a Breakthrough Therapy Designation (BTD) submission in the US and proposed EOP2 meeting request submission to the FDA. This will support a pivotal study program of neurodegenerative disease. The sponsor has also requested ICON’s clinical and regulatory support for a study in a new indication.