Amryt On Targeting Rare Disease Space In The Middle East
Executive Summary
The rare and orphan drug company Amryt speaks to the Pink Sheet about how to do business in the Middle East and why seeking marketing authorization could be a better long-term strategy than catering for named patient requests.
Amryt Pharma, a company with ambitions to become a leader in the rare disease space, has set its sights on the Middle East. David Allmond, the company’s chief commercial officer spoke to the Pink Sheet about the advantages of marketing authorization, the benefits of local partners and how to get the value proposition right for the payer.
Lojuxta (lomitapide), a treatment for adults with homozygous familial hypercholesterolemia (HoFH), a rare and life-threatening condition that impairs the body's ability to remove LDL cholesterol from the blood, is Amryt’s most important asset and is the focus of its Middle East strategy. Lojuxta is already approved in the EU and the US. Amryt licensed the drug from Aegerion Pharmaceuticals for markets in Europe, North Africa and the Middle East.
The potential demand for Lojuxta in the region is greater than it is elsewhere in the world, largely because of higher levels of consanguinity, which leads to a bigger incidence of genetic conditions, like HoFH, according to Allmond. “In one institution in Riyadh, Saudi Arabia, there are more patients identified with HoFH than we see currently in the UK,” he says.
To Register Or Not To Register?
Lojuxta is unlicensed in the region and provided on a named patient basis, which means that Amryt cannot actively promote the drug. However, it is able to provide disease education to physicians, explains Allmond. As such, the company’s presence is based on physicians making access requests for their difficult to treat patients that have exhausted all other treatment options, which have been made in Saudi Arabia, Oman, Qatar, Kuwait and most recently the United Arab Emirates, though the company does not divulge where patients are presently accessing the drug.
However, Amryt is currently examining whether to seek marketing authorization for Lojuxta in the region, either country by country, starting with the Saudi Food and Drug Authority, or through a pan Gulf Cooperation Council process that would give access to the product in all member states.
Abridged regulatory pathways have been established in Kuwait, Saudi Arabia, the United Arab Emirates, Jordan and Egypt. They offer companies with new products approved in the US, the EU or both quicker access to the market. (Also see "How Middle Eastern Markets Are Transforming Drug Reviews And Driving Competitiveness " - Pink Sheet, 22 Feb, 2018.) However, it is unclear at the moment whether Lojuxta could benefit from these arrangements because they require that the product under review has won their EU/US approval within a certain timeframe – Lojuxta was approved in the EU in 2013 and the US in 2012. Nevertheless, the situation is not black and white, explains Allmond. Because the product is an orphan serving high unmet need, there may be room for negotiation.
The decision of whether and where to file for marketing authorization depends on the cost, time and impact on the company, says Allmond. Although the current strategy of supplying on a named patient basis is working well, registration has benefits. For example, the company would be allowed to promote Lojuxta and therefore develop business further. There is also better regulatory protection over the long term.
“I think [registration] is the direction of travel but I’m not being hard and fast on it until we make the firm strategic decision of when and how to go. We are seeing a lot of access requests and seeing a lot of potential and we will meet that demand in the meantime. It is not as if it is immediately urgent to be in business. We are already in business,” says Allmond.
Pricing
The price for Lojuxta in the region is not far off European list prices, says Allmond. However, the company is prepared to negotiate on this on a case by case basis, but any agreements remain strictly confidential, he says.
Funding for the drug comes from governments, though for now, negotiations are conducted with specialist hospitals, which is where the patients are treated. However, there could be potential for talks with health ministries for national funding, if the demand for Lojuxta grows across broader institutions, says Allmond.
Generally, the challenge of convincing payers to fund an orphan drug is similar to that in other areas where the company operates, says Allmond. “Payers must understand where the drug fits in the treatment pathway and that there is significant unmet need with no adequate alternatives for patients. They need to understand that budget impact is manageable so the institution will get value for money and that there are no uncontrollable costs with a large patient population where the per capita price would become unmanageable.”
Nevertheless, one big difference in this region is the comparators. “This means that the way the company pitches the value can differ,” says Allmond. For example, in Saudi Arabia some patients are sent to the US for a liver transplant, which means a big outlay of costs for payers. In addition, within the region there is a smaller provision of apheresis, a process for removing lipids from the blood. “That increases the pool again for us because we have a pharmaceutical modality that may give a more immediate treatment alternative,” he says.
Expansion
To capitalize on the region’s opportunities, Amryt has partnered with local companies that can offer umbrella support as well as good knowledge of local customs and language. “To navigate the system, there is no one better than locals.” We also have a small dedicated Amryt team based in and around the region to provide necessary support to our partners.
The company has recently signed four distribution agreements for Lojuxta to further build its presence in the Middle East. The companies are Al Hafez Trading Establishment in Kuwait, Ebn Sina Medical in Qatar, Muscat Pharmacy and Stores in Oman, and Goro Healthcare in the United Arab Emirates and Bahrain.
Meanwhile, operations are a little different in Saudi Arabia, the biggest market in the region and as such has one of the biggest pools of patients. A mixed model through which Amryt runs its own team on the ground, but under the umbrella of a partner means that it can place a daily focus on the market but with a steer from a local partner who could facilitate interactions with stakeholders and distribution, explains Allmond.
Beyond Lojuxta, the company is keen to develop its presence in the region and build capability to serve other rare disease patients. This could include the company’s pipeline drugs as well as drugs licensed in from other companies. Indeed, Allmond says the company is always keen to seek additional assets in the rare disease space where there is high unmet need and believes Amryt is well placed to help other organizations navigate the complex landscape.
Amryt’s lead development asset is AP101, for the treatment of epidermolysis bullosa (EB) and which has recently won a Rare Pediatric Disease designation from the US Food and Drug Administration. The earlier stage product AP102 is being developed as next generation somatostatin analogue peptide medicines for patients with rare neuroendocrine diseases. And AP103 is a pre-clinical gene-therapy platform technology with potential to treat potential patients with recessive dystrophic epidermolysis bullosa, a subset of EB.
From the editors of Scrip Regulatory Affairs.