预约演示

Mirum scores 2nd FDA nod for rare liver disease drug Livmarli, this one to treat PFIC

2024-03-13

交易

上市批准并购临床3期临床2期申请上市

Mirum Pharmaceuticals' Livmarli has now been approved to treat two rare liver diseases, progressive familial intrahepatic cholestasis and Alagille syndrome, matching the two FDA approvals Ipsen has for its similar treatment Bylvay.

Mirum PharmaceuticalseaLivmarli42 million during its second full yerare liver diseasesirprogressive familial intrahepatic cholestasisent iAlagille syndrome Alagille syndromestart in fulfillingFDAs blockbuster potential.Bylvay

On WeLivmarli the company took another step toward reaching that goal as it secured aMirum Pharmaceuticalsvmrare liver diseaseestatic pruritus in patients ages 5 and older with progressive familial intrahepatic cholestasis (PFIC).

Combined with an FDA green light in 2021 to treat cholestatic pruritus in patients agesFDAmonths and oldLivmarliAlagille scholestatic pruritusarli (maralixibat) is now availableprogressive familial intrahepatic cholestasis (PFIC)ver diseases that cause pruritus, the intense itch that accompanies the genetic disorders.

“When we started FDA, we had this really intriguincholestatic pruritussome kids that did really well,” CEO ChrisAlagille syndrome Alagille syndrome (ALGS)iew with Fierce Pharma. “It feels good to get to the finish line for one of the kpediatric liver diseasesdo when we fpruritusnd started Miritch

Founded in 2018, Mirum acquired the rights to maralixibat from Shire that same year. Shire, for its part, acquired the drug through its Lumena buyout in 2014. A decade ago, the drug was tipped as a potential blockbuster when Shire paid $260 million up front for the mediciMirum

Now, the FDA apprMirumgives Foster City, Califmaralixibat Mirum a potential edge in its PFIC market rivalry with Ipsen. While Ipsen’s drug Bylvay (odevixibat) was approved in 2021 for PFIC and can be given to children as young as 3 months old, Livmarli’s approval is for a higher dose, which provides superior itch relief and bile acid clearance from the liver.

“(It) comFDAwith a great response rate and depth of response for PFIC patients,” Peetz said. “It’s a great new trIpsennt alterIpsene for paBylvay todevixibat aren’t responding or aren’PFICing fully controlled with current therapies."Livmarli itch

The next order of business for Mirum is to secure a label expansion to treat younger PFIC patients with Livmarli. The company has submitted a supplemental new drug application to introduce a higher concentration formula of Livmarli, which was used on some patients during the phase 3 MARCH study that paved the way for the PFIC approval. Peetz expects that green light to come later this year.

PFIC and ALGS cause a buildup oMirume acid in the liver, requiring patients to undergo transplants to stLivmarli. Life expectancy is shorter with PFIC, with only 50% of patients making it past age 10.Livmarli

Livmarli and Bylvay are both ileal bile acid transporter (iBAT) inhibitors which are taken orally. They are the only treatments approved for PFIC and ALGS, and that's expected to remain the case for years. Products in the works by potential competitors remain in early stages of development.

Livmarlias deBylvayd by Astraileal bile acid transporterch was bought out by Ipsen for $952 million in January of last year. Bylvay sales reaPFIC $73.ALGSllion for the final 10 months of the year, after the Ipsen acquisition was closed.

Bylvay competition in thAstraZenecaiatric liver disease which causes pruritisIpseniary atresia (BA), Ipsen is in phase 3 testing while Mirum has struggled in phase 2. In December, the company revealed the faIpsen of the EMBARK trial in which Livmarli came up short in all primary and secondary endpoints following Kasai surgery in babies between the ages of 21 and 90 days old.

The procedure for BA patients repediatric liver diseasets at the basepruritislibiliary atresia (BA)es bile flow using a portion of the intestine.Livmarli

“What the study showed in both groups (Livmarli and placebo) is the surgery works really well when it works, so you’re not really adding much,” Peetz explained.

Mirum has another iBAT inhibitor in theLivmarlitages of development in volixibat. The drug has shown “great precedent data,” in reducing bile acid Peetz said.

Mirumly of last yeiBAT inhibitor iBATMirum acquired two approved bile acid drugs from volixibatherapeutics, ponying up $210 million upfront and $235 million in potential milestones for Cholbam and Chenodal. The drugs combined for sales of $103 million in 2022.