Ultragenyx has been on a growth trajectory — simultaneously, the company is looking to stay true to the vision and culture. As CEO, president, and director of Ultragenyx Dr. Emil Kakkis says it’s part of his mission to create a “next-generation” rare disease company. Under Kakkis’ leadership, the focus has been on addressing the needs of as many rare disease patients as possible while retaining the heart and soul of a startup — even as the company expands.

“We’ve built a global commercial group that is collaborative and aligned, with ideas coming from all departments, and we’ve managed to retain the view that hierarchy shouldn’t matter even as we’ve reached 1,100 people,” Kakkis says.

This approach has translated into drug development success.

In addition to three FDA-approved treatments, including Mepsevii, the first-ever drug for mucopolysaccharidosis VII, an autosomal recessive lysosomal storage disease, and Dojolvi, the first approved for fatty acid oxidation defect, the California-based company has a healthy pipeline of other candidates for genetic diseases.

While some of the drugs in development target ultra-rare conditions, Kakkis says the company has seen the impact these products have on these small patient populations.

“As an example, Dojolvi is a product that had been used at an academic center for 12 years treating fatty acid oxidation defect patients with success, but, because it’s an oil, it’s not really protected by patents,” Kakkis says. “Our view was that Dojolvi is very potent in preventing kids with long-chain fatty acid oxidation disorders from having heart failure and ending up in the hospital. Our study showed that patients given this oil had a 50% reduction in median number of events and days in the hospital. We treated dozens of patients in our emergency care and 80% of them recovered from what was a devastating situation. And now that we have the product approved, we have more than 300 patients on the treatment in the first year of launch.”

Here, Kakkis shares his perspective on the rare diseases space, what makes it unique and what it takes to lead a collaborative and productive organization.

PharmaVoice: What does leadership in the rare and ultra-rare disease space entail?

Dr. Emil Kakkis: There’s a lot more to leading a rare disease biotech company than just getting a drug approved. There is leadership on how the field operates, how patient groups connect, and there is much greater public scrutiny of what you're doing and what it means to people.

Sometimes a company is the only one working in a disease and that creates expectations, which, in turn, requires a connection. It is a privilege as well as a responsibility to serve the rare diseases community. It takes empathy and understanding to be connected to patients while in early development, understanding how what we do affects them, while finding a balance between hope and being practical.

Can you share some unexpected learnings along the way in developing products for ultra-rare patients and building Ultragenyx?

There is a view that every disease has a patient group, but what we’ve found is that some diseases don't really have a well-developed patient group. When we started research into X-linked hypophosphatemia, which has 50,000 patients, there was more of a beginner organization, while on the other end of the spectrum there is FAST, an organization that is committed to finding a cure for Angelman syndrome, and raises huge amounts of money for research and patient advocacy. Patient advocacy group strength depends on the random occurrence of a strong leader who has been affected by the disease.

Many people in the industry hunger for that feeling of being affiliated with a company that’s going to do the right thing. This was key when we were building the business, as I felt strongly about retaining our ethical foundation. It was heartening to discover so many people who felt the same and that has allowed us to be who we are, grow and keep that strong, focused culture that not just talks, but does what we need to do — whether it’s patient access, pricing, policies, access strategies, support for patient groups, or product development.

What makes Ultragenyx’s approach unique?

We have a simple approach to product development, which is that any treatment we develop must have potent biology in a disease that has serious unmet need. We also focus on designing development programs that are smart and fast, that are adaptive in design, that rapidly drive to the correct dose, and that result in a shorter time of development. And, we have developed a strong regulatory negotiating strategy, which assists regulators who are presented with information about a disease they have never seen before.

Then, once a product is approved, there are two things that are important: one is getting patients access and the other is patient monitoring. Our commitment in the U.S. is that any patient properly prescribed the drug who needs treatment will get treated one way or another. And, this includes giving them the drug if insurance doesn't cover it.

Finally, post-approval follow up is traditionally handled through a registry where patients are loosely followed. We have a disease monitoring program, which is similar to good clinical practice compliant protocols followed during phase 3. The drug is administered through the commercial route, and the protocol monitors the disease. Because the drug is managed commercially, we're able to incorporate a large number of patients in this quality program to gather long-term outcomes data.

How do you balance the needs of patients and their caregivers and the demands of investors?

Truthfully, patients and investors want something similar, which is a safe, effective drug launched as promptly as possible. The question for patients is accessibility to treatment once a product is approved. Our view is that we can accommodate investors if we can generate the revenue numbers, and we can help patients if we come out with a moderate price point and negotiate better access terms with payers. So, we make up the volume of revenue through broader access to the drug rather than a higher price and more limited access.

What’s the one thing you wish you knew when you started in this space?

It’s been a journey of learning. I thought I was going to be a professor and when I went into industry, I realized there is something called management training. What we have done is to offer a lot of management training with a whole department focused on organizational development.

It can be a struggle when you start out, and we recognize that. If people from other biotech companies call us for help, we will help them develop their drug. Our success is dependent on developing our drugs, but it's not dependent on us holding back knowledge from everyone else.