FDA approves first treatment for patients with rare lung disease

Recently, the U.S. Food and Drug Administration approved Gifraj to treat acute hepatodiasis in adults, a genetic disorder that can lead to the accumulation of toxic molybrene molecules formed during the production of hemoerin (which helps bind oxygen to the blood).
“ This build-up leads to an acute seizure, called a rickets episode, which can lead to severe pain and paralysis, respiratory failure, seizures, and changes in mental state. "When a patient receives multiple blood transfusions, there is a risk of iron overload, which affects many organs," said Dr. Richard Pazdur, director of the FDA's Center of Excellence in Oncology and acting director of the U.S. Food and Drug Administration's Office of Oncology at the Center for Drug Evaluation and Research. Until today's approval, the treatment only partially alleviated the persistent pain that characterizes these attacks. Drugs approved today can treat the disease by helping to reduce the number of attacks that disrupt a patient's life. The
Givlaari is based on the results of clinical trials in 94 patients with acute hepatic rickets. The patient received a placebo or Givlaari. Givlaari's performance is measured by the rate of seizures of rickets, which require hospitalization, emergency treatment, or intravenous infusion of hemohemolyte at home. Patients treated with Givlaari were 70 percent less likely to develop rickets than those treated with a placebo.
Common side effects in patients taking Givlaari are nausea and injection site reactions. Health care professionals are recommended to monitor patients' allergic reactions and kidney function. Patients should have regular liver function tests before and during treatment.
The FDA gives priority to this adaptation review and breakthrough treatment.
Reblozyl was also awarded the title of Orphan Drug and provided incentives to assist and encourage drug development for rare diseases.
FDA granted the approval to Prokinix Pharmaceuticals. （cyy123.com）