Global companies expand business in expensive orphan drugs

Pfizer's ATTR-CM treatment Vyndamax approved by the Korean health authority

Shares same substance with Vyndaqel approved in 2015, but has different indication

Healthcare reimbursement is an issue for the expensive drug with yearly expense at KRW 300 million

Vyndaql approved for the Korean market in 2015.

 

Shares the same substance with Vyndmax but indicated for different disease.

 

As global pharmaceutical companies are on to expanding businesses in drugs for rare diseases, the companies are now winning item approvals in South Korean markets.

 

Rare diseases have limited patient size and insufficient treatment options, but the drugs are expensive enough for the companies to seek for profit.

 

On Aug.

 

19, Ministry of Food and Drug Safety (MFDS) approved Pfizer Pharmaceutical Korea’s Vyndamax 61 mg capsule (tafamidis).

 

The medicine is indicated to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.

 

Although the patient size is small, the disease is known to develop a heart failure and have a high risk of death.

 

Prior to the drug, no other treatment option was approved.

 

Previously, Pfizer has received approval on another drug with the same substance—Vyndaqel 20 mg capsule (tafamidis meglumine).

 

However, its salt base, substance amount and indication are different.

 

Vyndqel is also indicated to treat a rare disease, transthyretin familial amyloid polyneuropathy (TTR-FAP).

 

In October 2018, Vyndqel was listed for healthcare reimbursement and priced at 141,900 won per capsule.

 

A yearly drug expense would add up to 51.79 million won.

 

According to IQVIA, Vyndaqel earned approximately 1 billion won last year.

 

Considering the high upper limit pricing of Vyndaqel, Vyndamax would also be another ultra high-cost drug.

 

When the U.S.

 

Food and Drug Administration (FDA) approved of the drug in May last year, Pfizer put forth a yearly expense of USD 225,000, or approximately 266 million won, for the listed price.

 

The company also announced they are in a negotiation with insurers.

 

A growing number of global pharmaceutical companies launching ultra high-cost drugs for rare diseases is seemingly making the Korean health insurance authority anxious.

 

Early this year, Novartis submitted an official approval application for a spinal muscular atrophy (SMA) treatment Zolgensma with yearly expense proposed at 2.5 billion won.

 

Currently designated as an orphan drug, Zolgensma is in process of getting approved with expanded user age group in Korea.