Pediatric Orphan Therapy, From Phase II to Phase III and Commercial Manufacturing

The Challenge of Scaling Orphan Drug Development for XLHED

A Swiss customer was developing an innovative pediatric orphan biologic therapy. The treatment, administered in utero, had the potential to become the first approved therapy for a rare genetic disease.

Moving from Phase II to Phase III and preparing for commercialisation posed major risks. With limited internal resources compared to large pharmaceutical companies, the customer needed a dedicated CMC partner to:

• Manage the technology transfer of a complex biologics process to a capable CDMO.

• Scale up from pilot to industrial GMP production with robust quality controls.

• Ensure uninterrupted clinical supply for a pivotal Phase III trial across Europe and the USA.

• Navigate FDA and EMA regulatory pathways, including accelerated designations.

• Build a credible CMC and clinical dossier strong enough to attract a commercial partner.

Without specialist support, the foundation risked delays, supply interruptions, and the inability to secure a pharmaceutical partner to bring the therapy to market.

3Biotech’s CMC Expertise Driving Phase III and Commercial Readiness

The customer turned to 3Biotech for our proven track record in guiding complex biologic therapies through late-stage development. We:

• Transferred manufacturing to AGC Biologics, optimising the bioreactor process, scaling from pilot to industrial batches, and implementing robust quality controls.

• Supported the pivotal Phase III EDELIFE trial, ensuring compliant packaging, stability, and regulatory readiness across Europe and the USA.

• Led the regulatory strategy, securing FDA Breakthrough Therapy designation (2020) and EMA PRIME status, both accelerating timelines.

• Enhanced programme value, enabling a licensing and co-development deal (2020) with a European mid-sized pharmaceutical organisation, which assumed global rights to develop, manufacture, and market the therapy.