Ipsen's Bylvay is officially launched in Korea

New Milestone in PFIC Treatment

Will serve as a new turning point in treating PFIC

First oral therapy for an ultra-rare liver disease, reimbursed from October 2025

Marks a major milestone in access for pediatric patients with limited treatment options...“Provides an alternative to liver transplantation…expected to improve quality of life for pediatric patients”

From the left: Professor Ji-hyu Seo (Gyeongsang National University Hospital), Professor Seok-hee (Asan Medical Center), and Professor Hong Ioh (Severance Hospital)

 Ipsen Korea is introducing a treatment for progressive familial intrahepatic cholestasis (PFIC), an extremely rare hereditary liver disease, to the domestic market.

 

This launch opens new hope for pediatric patients who previously had no treatment option beyond liver transplantation.

 

Bylvay has been selected as the first product under Korea’s “Simultaneous Review & Negotiation Pilot Program,” which streamlines approval and reimbursement procedures.

 

The drug has been covered by national health insurance since October 2025, representing a symbolic step forward in improving access to rare-disease therapies.

 

On October 18, Ipsen Korea held a press conference at Sofitel Seoul Lotte World to commemorate the product’s domestic launch.

 

Bylvay is the first oral treatment option for PFIC.

 

Approved in the US and Europe in 2021, it has since gained approval in major countries.

 

In Korea, it received approval in 2023 and became reimbursed starting this October.

 

Professor Hong Koh of the Department of Pediatrics at Severance Hospital stated, "Although Bylvay was selected as the first drug for the parallel pilot program, the process was far from smooth.

 

Discussions involving experts should begin at the initial approval stage.

 

That's the only way to shorten the review period.” I am pleased that Bylvay’s approval and insurance coverage have improved patients' access to treatment.

 

I hope the pilot program is institutionalized quickly so it applies not only to PFIC patients but also to other drugs.

 

Many drugs are left unapproved.

 

I hope these many treatments can reach children as soon as possible.“ ” Liver transplant isn't always the answer...use of Bylvay will increase" PFIC is a rare hereditary liver disease that impairs bile flow in the liver, causing progressive liver damage and liver failure.

 

PFIC is a very rare disease, with only about 30 patients known to exist in Korea.

 

Caused by genetic mutations, PFIC presents with key symptoms including jaundice, severe itching, and poor weight gain.

 

It is typically recognized during infancy or childhood.

 

Treatment may involve supportive care, medication, or surgery, but most patients ultimately require a liver transplant.

 

Bylvay works by inhibiting the intestinal bile acid transporter (IBAT).

 

Through selective inhibition of IBAT, it prevents the reabsorption of bile acids from the intestines into the liver and increases their excretion through the intestines.

 

This lowers the concentration of bile acids in the body and helps improve symptoms such as itching caused by cholestasis.

 

Professor Seak-hee Oh of the Department of Pediatrics at Seoul Asan Medical Center said, “When bile acid levels exceed 400-500, patients cannot sleep even for an hour.

 

In clinical practice, we observed these levels drop by half when using Bylvay.” “Liver transplantation isn't always the answer.

 

Deaths also occur even after transplantation.

 

Ultimately, the best approach for PFIC patients is to avoid liver transplantation altogether.

 

I believe Bylvay’s arrival could shift the treatment paradigm.” Bylvay reaffirmed its efficacy this year through long-term follow-up data from the existing pivotal studies PEDFIC 1 and 2.

 

In the clinical setting, 15 patients showed a response at week 24, 14 of whom experienced improvement in pruritus, 5 experienced decreased serum bile acids, and 4 patients achieved both response criteria.

 

A significant proportion of FIC1-deficient patients with PFIC Type 1 showed improvement in pruritus when taking Bylvay, and most patients exhibited this response regardless of serum bile acid levels.

 

Furthermore, the reduction in serum bile acids and improvement in pruritus persisted from baseline through Week 96.

 

Ji-hyun Seo of the Department of Pediatrics at Gyeongsang National University Hospital stated, “Persuading stakeholders and reaching consensus to introduce this high-priced overseas drug was no easy task.

 

The introduction of Bylvay represents a meaningful achievement where the efforts of the government, patients, and the pharmaceutical company bore fruit.

 

The value of Bylvay is immense simply because it allows children to avoid liver transplantation.”