What is Therapy for Hypophosphatasia and Its Cost?

Hypophosphatasia Treatment Guide - Therapy Costs in India (ERT & More)

Hypophosphatasia (HPP) is a rare genetic disorder that affects bone and tooth development due to defective mineralisation. It is caused by mutations in the ALPL gene. This results in the accumulation of substances that impair bone strength and growth. The cost of Hypophosphatasia therapy in India can range between Rs. 1.2 crore and Rs. 6 crore annually, depending on the patient's age, severity of symptoms, and required dosage.

What is Hypophosphatasia Therapy?

Until recently, there was no definitive treatment for HPP. However, the approval of asfotase alfa, a recombinant enzyme therapy, marked a breakthrough in managing this rare condition. It replaces the missing enzyme, which allows the bones to mineralise properly and improve physical function, especially in children and infants.

What is the Cost Breakdown of Hypophosphatasia Therapy in India?

The therapy cost largely depends on the patient’s weight, as the dosage is calculated per kilogram of body weight. Below is an estimated cost range for various treatment options:

Disclaimer: The cost can vary depending on hospital location, patient requirements, etc.

Who Needs This Hypophosphatasia Therapy?

Doctors prescribe asfotase alfa primarily to:

• Infants with life-threatening symptoms,like respiratory failure and poor bone development.
• Children and adolescents with skeletal abnormalities, pain, mobility issues, or short stature.
• Selected adults with severe Hypophosphatasia symptoms.

Early diagnosis and intervention are important factors to improve outcomes and prevent complications such as fractures, rickets, and mobility issues.

Why is Hypophosphatasia Therapy Expensive?

• The rarity of the condition: HPP is a rare disease,and it affects only 1 in 100,000 individuals worldwide. The high cost of drug development and limited demand contribute to the price.
• High dosage requirement: Since the therapy needs a high dosage and is ongoing, it requires a consistent and high supply of medication.
• Imported drug: Asfotase alfa is not manufactured in India and must be imported, incurring customs duty and shipping charges.
• Specialised care: The treatment of Hypophosphatasia involves regular monitoring, which needs a team of specialists.

Is the Therapy for Hypophosphatasia Worth the Cost?

While the cost is undeniably high, the potential life-saving and life-improving outcomes make it a valuable option for families dealing with severe forms of Hypophosphatasia. Clinical studies have shown:

• It improves bone density and development
• It helps to reduce fractures and pain
• This therapy helps with better growth outcomes in children
• It enhances mobility

However, affordability remains a major barrier to this therapy, and it is not available in many parts of India.

Hypophosphatasia therapy in India is an expensive but transformative option for patients with this rare condition. If someone is diagnosed with HPP, early consultation with a specialist in rare diseases and access to the right support network can make a significant difference.