Despite some uncertainties in the clinical evidence, the U.K.’s National Institute for Health and Care Excellence (NICE) recommended the routine use of Sanofi SA’s Cablivi (caplacizumab) to treat acute episodes of acquired thrombotic thrombocytopenic purpura (TTP) in adults and in teens that weigh at least 40 kg (about 88 pounds).
The Nov. 12 announcement of the recommendation came after Sanofi “improved” its commercial arrangement with the pricing authority, NICE said. As is its practice, the agency didn’t disclose the discounted price the Paris-based company offered.
While adding Cablivi to the current standard of care is expected to reduce long-term complications and the risk of death from an acute TTP episode, NICE said it’s unclear how big that reduction will be due to the limited data reported to date.
However, those uncertainties likely will be offset by benefits associated with Cablivi, including expected reductions in the need for scarce resources such as donated plasma or intensive care beds, NICE said.
Also this week, the agency issued its final draft guidance recommending Abbvie Inc.’s Venclyxto (venetoclax), marketed as Venclexta in the U.S., in combination with Genentech Inc.’s Gazyva (obinutuzumab) as a first-line treatment for chronic lymphocytic leukemia (CLL) in people with certain genetic abnormalities, such as a 17p deletion to TP53 mutation, and those for whom existing CLL treatments are considered unsuitable.
NICE recommended that other treatment-naïve patients be allowed to access the 12-month, chemotherapy-free CLL regimen through the Cancer Drugs Fund, so more evidence can be gathered on the regimen’s cost-effectiveness in that group.
WHO maps end of the road for some tropical diseases
The 73rd World Health Assembly endorsed a new, 10-year global road map Nov. 12 that targets neglected tropical diseases (NTDs) and encourages a shift from vertical disease programs to cross-cutting approaches based on the needs of patients and communities.
The World Health Organization (WHO) road map sets global targets and milestones to prevent, control, eliminate and eradicate 20 NTDs and disease groups by 2030. It aims at reducing, by 90%, the number of people requiring treatment for NTDs, eradicating dracunculiasis and yaws, and eliminating at least one NTD in 100 countries.
Among the diseases targeted for some level of control or elimination are leprosy, chagas, dengue, rabies, leishmaniasis, schistosomiasis and scabies.
MHRA updates import licensing guidance
The U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) Nov. 12 updated its guidance on applying for a parallel import license for drugs.
The guidance details the three categories for parallel import applications, discusses labeling and packaging, and explains the process of renewing the five-year license and making variations. It also discusses fees and inspections.