Global Acute Intermittent Porphyria Market - 2022-2029

Global Acute Intermittent Porphyria Market - 2022-2029

Market Overview

The global acute intermittent porphyria market size was valued at US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).

Acute intermittent porphyria (AIP) is a rare metabolic disorder characterized by partial hydroxymethylbilane synthase deficiency (porphobilinogen deaminase).

Market Dynamics

The factors influencing the global acute intermittent porphyria market are the increasing research and development and the growing prevalence of acute intermittent porphyria.

The increasing research and development is expected to drive the market growth in the forecast period

Acute intermittent porphyria (AIP) is a rare metabolic disorder characterized by partial hydroxymethylbilane synthase deficiency (porphobilinogen deaminase). Porphyrin precursors can build up in the body due to this enzyme deficiency. A mutation in the HMBS gene causes this enzyme deficiency, inherited as an autosomal dominant trait (only one HMBS gene copy is affected). On the other hand, a deficiency is insufficient to cause disease symptoms, and most people with an HMBS gene mutation do not develop AIP symptoms. Additional factors that cause symptoms to appear to include hormonal changes associated with puberty, certain prescribed or recreational drugs, excessive alcohol consumption, infections, and fasting or dietary changes. The increasing research and development and FDA approvals are expected to drive market growth. For instance, givlaari (givosiran) was approved by the FDA in 2019 to treat adult patients with acute hepatic porphyria, including AIP. The goal of Givlaari is to reduce the number of attacks that patients have. Another heme preparation that can treat people with AIP is Normosang (heme arginate). Normosang isn't available in the United States, but it's widely used in countries where Panhematin isn't.

Side effects associated with acute intermittent porphyria are expected to hamper the market growth

If the diagnosis has not been made and harmful medications have been given, AIP can be extremely dangerous. If the disease is recognized early on and treatment and preventive measures are implemented before severe nerve damage occurs, the prognosis is usually favorable. Although most patients' symptoms go away after an attack, some develop chronic pain. After a severe attack, nerve damage and associated muscle weakness can improve over months or even years. Mental symptoms can occur during attacks, but they are rarely long-term.AIP can be particularly dangerous if a proper diagnosis is not made, especially if unsafe medications that aggravate the disorder are given. If AIP is diagnosed before severe nerve damage has occurred and treatment and preventive measures are initiated, the prognosis is usually favorable. Although most symptoms go away after an attack, some people may develop chronic pain. A severe attack's nerve damage and associated muscle weakness improve over time, but it may take months for the damage to heal completely.

COVID-19 Impact Analysis

The COVID-19 pandemic has kept the medical community on edge. While the pandemic will undoubtedly have direct consequences for infected patients and their families and broad social and economic consequences, it is also necessary to investigate the pandemic's impact on patients with diseases other than COVID-19. Covid-19 has negatively impacted the acute Intermittent Porphyria market due to a shortage of drugs and delayed treatment.

Segment Analysis

Prophylactic Hematin Infusions segment is expected to dominate the market growth

Acute intermittent porphyria (AIP) is an autosomal hereditary disease caused by a dominant-negative mutation in the porphobilinogen deaminase (PBGD) gene, which accumulates potentially neurotoxic porphyrin precursors during heme biosynthesis. AIP patients often require emergency hospitalization due to severe abdominal pain and sympathetic nervous system overactivity (e.g., hypertension, palpitation, tachycardia).

Symptomatic treatment and suppression of hepatic rate-limiting heme biosynthesis enzymes are used to treat acute AIP attacks (i.e., 5-aminolevulinic acid synthase 1, or ALAS-1). In the case of mild attacks, glucose, which inhibits ALAS1 by affecting peroxisome proliferator-activated receptor-gamma coactivator1-alpha, could be considered. Intravenous heme is the most effective treatment for severe acute attacks because it provides exogenous heme and inhibits ALAS1 transcription, resulting in a rapid reduction in ALA and PBG overproduction. Acute porphyric attacks usually last 1–2 weeks and can be effectively treated with 3–5 days of intravenous heme therapy. PANHEMATIN is a hemin for injection used to treat recurrent attacks of acute intermittent porphyria in women who have a menstrual cycle after carbohydrate therapy has been proven or suspected to be ineffective.

Geographical Analysis

North America region is expected to hold the largest market share in the global acute intermittent porphyria market

The growing prevalence of acute intermittent porphyria and increasing FDA approval is expected to drive this region's market growth.

On 20th November 2019, the U.S FDA approved Alnylam Pharmaceuticals' investigational candidate, givosiran, marketed as Givlaari, to treat adults with acute hepatic porphyria (AHP). The FDA granted priority review status to the therapy's new drug application (NDA) for that indication less than four months ago. Hence, with the increasing FDA approvals, the acute intermittent porphyria market is expected to grow in this region.

Competitive Landscape

With mergers, acquisitions, and product launches, the global acute intermittent porphyria market is moderately competitive. Some of the key players in the market are Alnylam Pharmaceuticals and RECORDATI S.p.A

Alnylam Pharmaceuticals

Overview: Alnylam Pharmaceuticals Inc. is a biopharmaceutical company specializing in developing, commercializing, and discovering RNA interference therapeutics for genetically defined diseases. The company was established in 2002 and is based in Cambridge, Massachusetts.

Product Portfolio: Givosiran is an RNAi therapeutic that targets delta-aminolevulinic acid synthase 1 (ALAS1) and is used to treat acute hepatic porphyria (AHP). It is marketed as GIVLAARI in areas where it has been approved (givosiran). Acute intermittent porphyria (AIP), variegate porphyria (VP), hereditary coproporphyria (HCP), and ALA-dehydratase deficient porphyria are the four types of AHP (ADP).

Key Development: On 2oth November 2019, the U.S FDA aprroved Alnylam Pharmaceuticals' investigational candidate, givosiran, which will be marketed as Givlaari, to treat adults with acute hepatic porphyria (AHP).

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