Case Study: Nobelpharma achieves orphan drug dose optimization with Certara

Dose Optimization Using Population PK for an Orphan Drug

Nobelpharma, a Japan-based pharmaceutical company, faced the challenge of optimizing the dose for its orphan drug RAPALIMUS® for a new indication treating rare lymphatic diseases. Due to the small number of patients in the high-risk clinical trials, standardizing a dosage for the drug label using conventional methods was impossible. The company collaborated with vendor Certara to apply a population pharmacokinetic (PPK) modeling approach.

Certara’s consulting team established a population PK model using a non-linear mixed-effects model, which identified factors affecting the drug's pharmacokinetics. This enabled simulations to set doses based on body surface area and supported the regulatory submission. As a result, a new dosing recommendation was successfully included in the drug’s package insert, and post-marketing therapeutic drug monitoring was implemented. Certara’s solution was critical in gaining approval for the new indication where traditional clinical trials were not feasible.

Nobelpharma