At present, Zolgensma is being investigated in the global Phase III STR1VE clinical program (consisting of STR1VE-US, STR1VE-EU, and STR1VE-AP) to evaluate the intravenous (IV) formulation of AVXS-101 in patients who have SMA Type 1, and the multinational Phase III SPR1NT trial in presymptomatic patients who have a genetic diagnosis of SMA with two or three copies of the SMN2 gene. Additionally, AVXS101 intrathecal administration is being studied in a Phase I/II STRONG trial in patients who have SMA Type 2 and three copies of the SMN2 gene. The new data from trials were presented at 2019 congresses, including the American Academy of Neurology Annual Meeting.
The dynamics of the AAV-gene therapy market is anticipated to change as companies across the globe are thoroughly working toward the development of new AAV-gene therapy options to treat a wide array of indications. Key players, such as Biomarin Pharmaceutical, Roche (Spark Therapeutics), Sangamo and Pfizer and others are involved in developing AAV-gene therapy for indications such as Hemophilia A and B, MPS, and others.
In 2019, Luxturna was recommended by NICE, within its marketing authorization, as an option for treating RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. It was only recommended if the company provides voretigene neparvovec according to the commercial arrangement.
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Luxturna, received ‘considerable added benefit’ from G-BA, the German health technology assessment. Luxturna was launched in Germany in April 2019 at the manufacturer price of €345,000 per eye per patient. G-BA motivated their decision by Luxturna’s favorable clinical efficacy: improvement of functional vision/orientation, light sensitivity and perimetry. In addition, G-BA mandated a reassessment in January 2022 to evaluate Luxturna’s long-time safety and efficacy. The reassessment will be based on data from PASS, the post-approval safety study registry and an ongoing, one-armed observational trial, the 302 study. The reassessment request falls into the remit of GSAV, in August 2019 introduced law on better safety in drug supply.
In May 2019, ICER found Zolgensma to be cost-effective up to USD 5 million at the ultra-rare disease threshold of USD 500,000 per QALY and up to USD7 million when using life-years gained compared to best supportive care. In a report assessing Novartis’s Zolgensma, ICER stated that the drug’s USD 2.1 million price tag falls within the upper bound of ICER’s value-based price benchmark range.
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