Can you provide an overview of LFB’s presence in the US and the rPRO Technology powering the company’s development?

LFB US has its roots in a company that LFB acquired in the early part of the 2000s, which was a spinoff of Genzyme, called Genzyme Transgenics. The transaction brought the rPRO Technology into the LFB family, and out of that technology today we have two FDA approved products. One of them is Sevenfact, which is currently in the market, and the other is our first approved product called ATryn, which is on its way back to the market. With the rPRO platform, we have opportunities for the manufacturing and development of many other recombinant proteins, but we decided three years ago to focus specifically on the ones that have the highest chance of success and those are the two products that we have continued to commercialize. It is a clearly-focused strategy.

The rPRO Technology is a revolutionary approach to protein manufacturing, created to avoid the limitations of the existing method of conventional cell culture (CHO). rPRO can be defined as a more natural, scalable and cost-effective way to manufacture these recombinant proteins. Natural, because these proteins are expressed in animal milk; scalable because there is very little additional cost going from 5 kilos of protein to 500 kilos for example, enabling the manufacturing of commercial quantities at high expression levels in difficult to express proteins such as antithrombin.

What is the potential market size and unmet medical need Sevenfact is serving?

Sevenfact is indicated for Hemophilia A & B patients with inhibitors. It is a relatively small market, even though there is a substantial Hemophilia patient pool, once you go into the A & B patients with inhibitors, there are less than 1,000 patients in the US. It is a targeted market, with only one other product approved in the market, and that product has held a monopoly position for many years. Consequently, LFB had a very big incentive and was determined to bring an additional choice to these patients.

What are some of the keys to executing a successful product launch?

Launching Sevenfact in the middle of a pandemic was the ultimate challenge. A year after the FDA approval, the level of engagement has been positive. We are also in a much better place as it relates to COVID-19 and access to physicians and hospitals than we were six months ago. Nevertheless, the launch tactics and the preparation of the launch remains the most important part for successfully launching a commercial product. Being able to focus on high quality and timely execution of your preparation steps remains paramount. We have worked extensively on evidence generation, scientific communication, having a strong publication plan and KOL engagement. The digitalization of the Marketing and Sales strategies these days has also become an increasingly crucial element of executing a successful launch strategy.

To what extent do payors play a role in the commercial success of a product. How is Sevenfact positioned form that standpoint?

Development of a pharmaceutical product and getting FDA approval are important first steps. However, regulatory approval does not always equal commercial success. Commercial success, especially with products under orphan drug designation, is challenging.  Your work with payors needs to be very targeted and clear. Sevenfact is a product that breaks up a monopoly, so that is already a good starting point for discussions with payors. We are giving them a different option, and this is an opportunity to provide better value for patients and all stakeholders in general. Our commercial partner HEMA Biologics has held very successful conversations with payors; everybody wants to have multiple products in formulary. Now it is a question of which one provides better value so they have choices in the way the priorities are addressed.

Can you provide an update on ATryn’s potential reentry into the market?

ATryn is a product that was approved by FDA and EMA and it was in the market in the US between 2010 and 2015. Its BLA has been in an approved status since the original approval. Unfortunately, the previous drug substance contract manufacturer for ATryn faced significant challenges with FDA. That CMO ended up shutting down their facility, and ATryn was one of the products that suffered from that situation. We embarked on a technology transfer with another CMO, and the process to get them through FDA approval was finally achieved last year. This was an important milestone, but it does show that it is not an easy road. Currently, we are preparing the return of ATryn into the market. It is also a product that breaks a monopoly situation as there is only one other antithrombin approved product, which is plasma derived. ATryn is a recombinant protein, so there is very clear differentiation with all the advantages recombinant proteins have over plasma derived proteins. We are in the process now of looking for a commercial partner and we are looking forward to offering ATryn to patients and Doctors in need of this life-saving therapy in the near future.