According to BioSpace, a consulting firm called Fact.MR has conducted a study into the market for acute hepatic porphyria (APH). The point of this study was to understand the future of treatment for APH and other metabolic diseases. Fortunately, Fact.MR found that there is a positive outlook for this condition in 2021 and well into the future.

About Acute Hepatic Porphyria

APH falls under the larger group of porphyria, and it occurs when the body lacks an enzyme needed to produce heme, a part of hemoglobin. This leads to an accumulation of porphyrins and porphyrin precursors, which then results in oxygen depletion. Looking more specifically to APH, this disease pertains to the liver, and it can be further divided into subcategories as well. It is characterized by attacks on the organs in the large cavity of the torso and parts of the nervous system. These attacks cause symptoms such as muscle weakness, convulsions, anxiety, irritability, abdominal cramping, confusion, sensory loss, and auditory or visual hallucinations. In terms of triggers, stress, medications, alcohol, hormonal changes, a diet too low in calories, and infections can all bring on an attack. To treat these symptoms, doctors focus on pain management, reversing attacks, and preventing any neurological symptoms. Hemin is often given through an IV, as is glucose if necessary. In severe cases, one may require a liver transplant.

Fact.MR’s Study

One of the first things the study focuses on is the incidence of APH. The National Center for BioTechnology Information places the global incidence at one of every 500 to one of every 50,000 people. Due to this high incidence, Fact.MR asserts that there will be a higher demand for treatments and research in the upcoming years.

We are already seeing the development and approval of treatments for APH, with Givosiran receiving FDA approval back in 2019. This trend is expected to continue as manufacturers see the worth in developing drugs for metabolic diseases like acute hepatic porphyria. Other results of the study include:

• IV administration of hemin will continue as the main treatment option
• Swift approval from the FDA gives the United States the chance to be a market for AHP
• Hospital pharmacies will be some of the largest distributors of treatments
• Acute intermittent porphyria is projected to be the most commonly diagnosed subtype of AHP
• Germany and France have increased healthcare spending, giving them the chance to expand their AHP market
• The UK should see more research on diagnosis in asymptomatic manifestations of the disease
• Government funding for research will propel AHP research forward
• Improvements in the diagnosis of metabolic diseases will spur further AHP research
• More regulatory approvals are a good sign for future drugs

The study also identified a number of restraints on the AHP market, one of which was limited awareness of AHP. Another is limited access to healthcare, which prevents proper diagnoses and treatment. The third obstacle that they identified was the asymptomatic manifestation of the disease, which often results in delayed diagnosis.

Looking Forward

A number of players have already entered the AHP market, namely Alleviare Life Sciences, Recordati Rare Diseases, Alnylam Pharmaceuticals, and Lundbeck Inc. These companies focus on improving the understanding and lives of those with AHP, from creating treatments to platforms that advance comprehension. In addition, some of these companies are partnering with government initiatives to help patients.