Commercial PreparednessCompany has built commercial infrastructure with rare disease sales specialists, medical science liaisons, and field access staff to support patient treatment starts at priority accounts, indicating readiness for market launch.
Regulatory PathwayRegulatory alignment on using reductions in the biochemical marker PPIX as a surrogate endpoint supports a clearer path to accelerated approval for the oral therapy.
Safety ProfileExtensive Roche-derived safety data and prior development studies show no signal of abuse risk, reinforcing the drug's favorable safety profile for regulators and prescribers.