Introduction/Background

A biotech company targeted a new oncology indication with an improved delivery technology for an already approved drug. Because the active pharmaceutical ingredient had previously been approved, the company believed it could leverage the 505(b)(2) pathway to seek FDA approval for the new indication — but issues with the FDA threatened to derail the project.

Seeking to tap in-house drug development experience, the biotech company began nonclinical trials. Because there is no standard approach to achieving a 505(b)(2) NDA, the client’s in-house team began having trouble addressing multiple issues with the FDA. It feared that one wrong move could cause permanent damage to the program.

Solution

When the client approached Premier Consulting, we leveraged our longstanding relationship with the relevant FDA division and developed a strategy that would enable an efficient path to approval. We also compiled evidence demonstrating the safety and efficacy of the drug. Our cross-functional teams examined the project from every angle with the goal of program optimization. We corrected and rewrote the deficient modules, turned the nonclinical data into a meaningful story covering essential pharmacokinetics, toxicology, nonclinical testing strategy, pharmacodynamics, and toxicokinetics, and compiled relevant nonclinical study reports.

Takeaway

Properly assembled, the NDA package showed outstanding results and revealed patients’ unprecedented success with the drug, leading to remission and longer lives. The biotech company, able to present a high-value, viable, and realistic product with imminent regulatory success, was acquired by a larger company for a substantial sum.

Outcome

Having demonstrated its viability, the biotech company was acquired by a larger company. The FDA subsequently approved the drug, which has the potential to become a new “gold standard” treatment.