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ACPs to lead gene therapies in haemophilia market growth

New therapies to hit existing players


Roche’s Hemlibra and Alnylam’s late-stage candidate fitusiran are likely to seize a bigger share of an expanded haemophilia market than gene therapies, according to a new analyst report.

GlobalData predicts the wave of novel treatments for haemophilia A and haemophilia B market will see the market grow from $6.93bn in 2018 to $9.29bn in 2028.

That means even though potential gene therapies for both haemophilia A and B could offer patients a cure, many payers and clinicians are predicted to opt for long-term treatment with Hemlibra and fitusiran instead.

The two drugs have distinct mechanisms –  Hemlibra is a bispecific factor IXa- and factor X-directed antibody, while fitusiran is an RNA interference (RNAi) therapeutic targeting the endogenous anticoagulant antithrombin (AT) – but have been given the umbrella term ‘alternative coagulation promoters’ (ACPs).

Hemlibra was launched in the US in late 2017 and in Europe in 2018, and has been a runaway success, and is now available for haemophilia A patients with and without inhibitors.

Fitusiran, which Alnylam is co-developing with Sanofi, is in phase 3 trials for hemophilia A and B patients with or without inhibitors, and is expected to arrive on the market in 2021.

GlobalData says the growth rate in the sector will be a modest Compound Annual Growth Rate (CAGR) of 3%,reflecting what is likely to be a cautious adoption of new therapies for patients with the difficult-to-manage bleeding conditions.

Replacement factors are the current standard-of-care, representing 97% of the total sales of the haemophilia market in 2018. However, their market share is expected to decline to 70% by 2028 thanks to the arrival of alternative coagulation promoters (ACPs) and gene therapies.

The new therapies target significant areas of unmet patient need, such as reducing the need for intravenous infusions and minimising the risk of developing neutralising antibodies, or inhibitors, against replacement therapies.

Tajekesa Chapman, PhD, senior oncology and haematology analyst at GlobalData commented: “ACPs and gene therapies are the most anticipated drug classes for the treatment of haemophilia A and B as they will provide more effective treatments for patients with inhibitors, more convenient administration routes and less frequent dosing, and, in the case of gene therapies, provide a potential cure for the disease.”

GlobalData found that key opinion leaders in the eight major markets (the US, France, Germany, Italy, Spain, UK, Japan, and China) are highly optimistic about these therapies and agree that they will drive change in the treatment landscapes for haemophilia A and B.

Chapman says Hemlibra and fitusiran  will achieve combined revenues of $1.67bn in 2028l, and will represent 18% of total haemophilia drug revenues in 2028 in the eight major global markets.

BioMarin’s ValRox, which is expected to launch as early as 2020, and Spark Therapeutics’ SPK-8011 for haemophilia A, and uniQure’s AMT-061 and Pfizer/Spark Therapeutics’ SPK-9001 for haemophilia B, are leading the clinical development race for gene therapies.

The analysts anticipate the total revenues from these four therapies to be around $1.15bn in 2028, which will make up 12% of total haemophilia drug revenues in the eight major markets.

Chapman says this crowded field will inevitably create competition.

Chapman concluded: “Gene therapies are positioned for a limited patient population, such as severe adult haemophiliacs, previously treated patients, and patients with no history of inhibitors, and questions have already been raised about their limited long-term safety and efficacy data. Therefore, first-to-market advantage will drive commercial success.”

BioMarin currently has the edge on its rivals in this race to market, having recently announced that it will file ValRox in the US and Europe in Q4 this year.

Article by
Andrew McConaghie

16th July 2019

From: Marketing



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