Suspicion of data manipulation in the world's most expensive orphan drug

The FDA started its investigation on the grounds of data-manipulating incidents in the studies of the gene therapeutical drug "ZOLGENSMA®" (onasemnogene abeparvovec-xioi), a treatment for spinal muscular atrophy (SMA type I-III) in young children. The cost of a single injection is 1.9 million euros, which makes it the most expensive drug in the world.

The medical background of this rare disease can be traced back to a bi-allele mutation in the ''survival motor neuron 1'' (smn1). The dysfunction in the gene causes the loss of the gene product as well as the successive loss of crucial nerve cells in the central nervous system (spinal cord and brain stem), followed by muscle weakness as well as muscle atrophy in the extremities near the trunk. The promising AAV-based gene therapy showed positive effects in the STRIVE and START studies in terms of survival and motor skills development.

Novartis allegedly delayed informing the FDA about the data manipulation of a non-clinical sub-study. Consequently, the world-renowned company risks criminal sanctions and hence a reputational damage. In any case, the stock market price temporarily fell by more than 4% after the news went public.

However, the FDA sees no reason to doubt the clinical efficacy of the drug and has not restricted its distribution and use in the American market yet. The ongoing market access authorization procedures for Zolgensma® in the EU and Japan have not been affected.

The professional consultants from SKC consulting are supporting pharmaceutical companies at the market access of their innovative drugs. Further, SKC provides appropriate solutions for the reimbursement dilemma. Please find detailed information in our publication "Gene therapy ante portas - appropriate solutions for the reimbursement dilemma."

Sources:
Reuters: Manipulation von Testdaten waren Einzelfall
Center Watch: Zolgensma
FDA: FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy
Mitteldeutscher Praxisverbund Humangenetik: Spinale Muskelatrophie (SMA)
Krankheitsbilder Spinale Muskelatrophien
Frankfurter Rundschau: Novartis-Medikament Zolgensma