The first type 1 hepat/nephrosyndrome (HRS-1) drug was recommended and approved by an FDA panel of experts.

On July 15, biopharmaceutical company Mallinckrodt announced that the FDA's Advisory Committee on Cardiovascular and Kidney Medicines had voted (8 in favour of vs 7) to recommend approval for its use in the research drug terlipressin for the treatment of adults with hepatic and kidney syndrome type 1 (HRS-1).
HRS-1 is an acute and life-threatening syndrome involving acute renal failure in patients with cirrhosis.
it can lead to life-threatening kidney failure within a few days and can only be addressed through liver transplantation and its effects on kidney function.
the disease often requires exclusion from diagnosis and is often difficult to diagnose in a timely manner.
without treatment, the median survival time in HRS-1 patients was about two weeks, with a mortality rate of more than 80 percent over three months.
the disease is estimated to affect 30,000 to 40,000 Americans each year, but there are currently no approved treatments in the United States. "In many countries where terlipressin is approved, the drug combined albumin is recommended for HRS-1 treatment standards," said Francois Durand, M.D.,
, on behalf of the International Astral Water Association.
HRS-1 is one of the most serious complications of end-stage liver disease, and the Advisory Committee's vote in favor gives hope to American patients who need treatment.
" Terlipressin is an effective selective pressure-based classier analogue for V1 receptors, and the drug has been listed in countries such as Europe and Australia, and approved adaptations include the treatment of deseptic deseptic shock, liver and kidney syndrome, and ruptured varicose esophagus blood canals.
terlipressin molecular structure In April, the FDA accepted an NDA review application for the drug, based primarily on positive results from Phase 3 clinical trial SAKIT.
the largest-ever prospective study to assess the safety and efficacy of terlipressin in HRS-1 patients, with 300 patients enrolled in the U.S. and Canada, and the study reached the main endpoint: terlipressin reversed HRS-1 in 29 percent of patients, compared with a placebo reversal rate of 16 percent.
"reversal" means reducing the patient's creatinine level (supported by hemodialysis to the kidneys) below a certain threshold and keeping the patient alive for at least 10 days without renal replacement therapy (RRT) such as hemodialysis and kidney transplantation, but the drug is not curable. the
CONFIRM trial also showed that the risk of terlipressin included respiratory failure (10%) and the placebo group affected 3%.
9 percent of patients in the treatment group died of respiratory failure, 6 percent from sepsis shock and 3 percent from sepsis, compared with 1 percent, 2 percent and 0 percent in the placebo group. "I voted against it because of the evidence that the alternative endpoints were benefited from the trial," said Thomas Cook, a professor in the Department of Biostatistics and Medical Informatics at the University of Wisconsin-Madison,
.
Given the flawed way pharmaceutical companies analyze data, I see no evidence that alternative benefits translate into meaningful clinical gains, and I see no evidence that the potential for such benefits can offset the risk."
" those who voted for it stressed the seriousness of HRS-1 and did not approve the therapy. Paul Ridker, a cardiologist at
Brigham and Women's Hospital, said: "I voted yes, though a little reluctantly.
because for a serious disease, there is a little more therapeutic than no.
" FDA designated the prescription drug user costs act (PDUFA) target date of September 12, 2020.
while the Advisory Committee's recommendations are not binding, the FDA generally prefers to follow the committee's recommendations.
but the most recent exception is Akcea Therapeutics' antisense nucleic acid drug Waylivra (volanesorsen), which the FDA rejected in the context of the Advisory Committee's approval of its use for familial celiac microcephaly syndrome by a large percentage (12 vs 8) because it allows patients to reduce the risk of platelets and reduce the risk of uncertainty and the benefit/risk ratio.
Source: 1, Mallinckrodt's Kidney failure med squeaks by FDA Panel in 8-7 Vote 2, Mallinckrodt Announces U.S. Food and Drug (FDA) AdvisorY Committee Voted to Recommend Terlipressin for Approval to Patient S.