Santhera Pharmaceuticals announced the complete worldwide divestment of Raxone (idebenone) to Chiesi Farmaceutici while still being eligible for milestone-based payments from the drug sales.
In 2019, Santhera and Chiesi signed a global licensing agreement, except in North America and France, for Raxone to treat Leber's hereditary optic neuropathy (LHON) and any other ophthalmological conditions.
Chiesi will also inherit the financial responsibility of the €25.3m ($27.9m) reimbursement agreement of Raxone in LHON with French reimbursement authorities, as per the press release.
Since August 2021, and prior to finalising a reimbursement agreement with the French Government in February 2023, Santhera was providing Raxone for free to treat LHON. Drug sales resumed in April 2023, as per unaudited annual results released the same month.
Following the latest news, Santhera would be eligible for US sales milestone payments for Raxone for up to $10m, and other milestone payments if Chiesi expands the use of Raxone in non-ophthalmological indications.
Santhera expects to maximise the reduced financial burden of the Raxone development programme to focus on the vamorolone’s European launch.
In September 2020, Santhera obtained the global rights of vamorolone in Duchenne muscular dystrophy (DMD) from ReveraGen BioPharma. Following this, Santhera signed a licensing agreement with Catalyst Pharmaceuticals and Sperogenix Therapeutics. This involved giving Catalyst licensing rights in the US, Canada, and Mexico for a $90m upfront payment, along with future milestone-based payments, and licensing rights in China to Sperogenix for $124m, including upfront and milestone-based payments.
Vamorolone has a US FDA’s Prescription Drug User Fee Act (PDUFA) date for approval in DMD on 26 October 2023. Marketing authorisations for vamorolone are also under review with European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).
Another drug in Santhera’s pipeline is lonodelestat, a selective peptide inhibitor of human neutrophil elastase, which has orphan drug designations in the EU and US for treating cystic fibrosis, alpha-1 antitrypsin deficiency (AATD), and Primary ciliary dyskinesia (PCD).
