“A new long-acting treatment (‘Qfitlia’ or fitusiran) for hemophilia was approved. Patients tend to rely on regular infusions of clotting products or similar products to prevent excessive bleeding. This new treatment..is given by injection roughly once per two months. In phase 3 trials, it reduced bleeding episodes by around 70% more than standard treatment,” reports The Doomslayer.
The FDA explains why it approved the new treatment:
Qfitlia is administered under the skin (subcutaneously) starting once every two months. The dose and frequency of injections are adjusted using the FDA-cleared INNOVANCE Antithrombin companion diagnostic test. This companion diagnostic is intended to monitor and—by informing dosing and frequency of injections—achieve antithrombin activity in the target range to reduce the risk of bleeding and to reduce the risk of excessive blood clotting. The FDA granted clearance of the INNOVANCE Antithrombin test to Siemens Healthcare Diagnostics GmbH.
Qfitlia’s efficacy and safety were assessed in two multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male patients with either hemophilia A or hemophilia B. In one study, participants had inhibitory antibodies to FVIII or FIX and previously received on-demand treatment with medicines known as “bypassing agents” for bleeding. In the second study, participants did not have inhibitory antibodies to FVIII or FIX and previously received on-demand treatment with clotting factor concentrates. In the two randomized trials, participants received either a fixed dose of Qfitlia monthly or their usual on-demand treatment (bypassing agents or clotting factor concentrates) as needed for nine months. The fixed dose of Qfitlia is not approved because it led to excessive clotting in some patients.
In other news, the first non-opioid painkiller in decades was developed for surgical treatment. It has fewer side effects than other painkillers and doesn’t seem to be addictive.
Malaria vaccines are saving thousands of lives in Africa.
Last year, a treatment was discovered for sleeping sickness, a disease that kills 50,000 to 500,000 people per year. Sleeping sickness is an awful disease that begins with fever and aches. Then things get worse. The parasite that causes the disease will disrupt sleeping patterns and cause aggressiveness and psychosis. The death rate from sleeping sickness is close to 100%, if it is untreated.
The customary treatment for sleeping sickness was also quite awful: a drug that is toxic to the kidneys, followed by an arsenic-based drug that’s toxic to the brain. The treatment kills up to a tenth of all patients. But thanks to a recent discovery, people can take the drug fexinidazole to treat the disease instead.
For many years, therapy would begin with a lumbar puncture to see if the parasite has invaded the central nervous system, followed by injections of a drug that damages your kidneys, followed by an arsenic-based drug that harms your brain. But scientists have discovered that fexinadazole can be used to treat sleeping sickness, first the chronic variety, and more recently for acute sleeping sickness as well. European regulators approved the drug based on results from a clinical trial showing that a 10-day course of pills cures acute sleeping sickness in 97% of patients. That will lead to approval of the drug by countries outside of the EU, many of which more or less automatically approve drugs approved by the European Medicines Agency.
