Two drugs have PDUFA dates in May

Sanofi’s dengue vaccine (Dengvaxia) has a PDUFA date of May 1, 2019.  Dengvaxia is a 3-dose, subcutaneous vaccine for the prevention of malaria. The vaccine was tested in Brazil and the Philippines and was able to prevent 2/3 of cases and prevented 8/10 hospitalizations.  Two large trials found efficacy of around 60%. Two Phase III pediatric trials demonstrated vaccine efficacy of 56.5% and 60.8%, respectively, against symptomatic virologically confirmed dengue. The vaccine appeared to be more effective in older children possibly due to higher baseline seropositivity and potentially a more broadly protective immune response. A four-year follow-up of two Phase II trial involving 1,378 patients found the vaccine demonstrated long-term immunogenicity. Dengvaxia is approved in 5 countries, which include Brazil, Philippines and Mexico. If that patient contracts the viral infection naturally, their immune system could process it as a second infection that is much more severe.  The FDA has granted Dengvaxia a priority review. An analysis by WHO cautioned of an increased risk for severe dengue in seronegative vaccine recipients compared to seronegative non-vaccinated patients, but long-term protection in seropositive individuals is achieved. The FDA’s Vaccines and related Biological Products Advisory Committee voted to recommend approval for Sanofi’s Dengue vaccine for patients 9 to 17 years, but not in adult patients.

Novartis onasemnogene abeparvovec (Avexis) has a PDUFA date for an unspecified date in May 2019. Onasemnogene abeparvovec is a single dose, intravenous Infusion for the treatment of spinal muscular atrophy. Onasemnogene abeparvovec was evaluated In the 20-month, 15 patient, Phase I, START trial, where treatment with the gene therapy resulted in all patients being alive compared to 8% of a historical control group of patients with SMA type 1. At 3-months there was a 15.4 point improvement in the CHOP INTEND score of motor function score in patients treated with onasemnogene abeparvovec compared to a decline in the historical control. Novartis announced interim data from 22 patients in the Phase III, STR1VE trial, where treatment with onasemnogene abeparvovec resulted in a 11.8 point improvement in the CHOP INTEND score at 3 months in patients with SMA type 1. One patient died from respiratory failure, which was deemed to be unrelated to treatment. When approved onasemnogene abeparvovec will compete with nusinersen. Novartis indicated it may price the gene therapy as high as $4-5 million. Using a placeholder cost of $2 million, ICER estimated a cost per QALY for onasemnogene abeparvovec of $243,000 compared to best supportive care. ICER estimated that to be cost effective the cost of onasemnogene abeparvovec would need to be $900,000 to be in a $150,000 of QALY range, or $1.5 million if estimating the cost for each additional year of life gained (LYG) of $150,000.