In the past few years, the FDA has set its sights on fast-tracking scores of new medications to more quickly and efficiently get needed treatments on the market. All told, the agency approved 59 new molecular entities in 2018 — a significant jump from the 47 approvals in 2017.
Among the approvals slated for this year (or early 2020) is a wave of gene therapies and anti-inflammatories that are poised to reach blockbuster levels by 2024.
According to a report by EvaluatePharma, an analysis firm that provides forecasts for the biotech and pharma industries, the top 10 drugs expected to win approval this year will collectively pull in $18.21 billion in the next five years. Here are the drugs with the highest sales potential.
Although the patient population intended for Ultomiris, a next-generation version of Alexion’s blockbuster Soliris, is small, EvaluatePharma predicts that its sales will be major. In December, the FDA approved Alexion’s drug to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a life-threatening disease that can cause blood clots, impairs bone marrow function and breaks down red blood cells. Only about 500 Americans are diagnosed with PNH each year, but sales for Ultomiris are expected to hit close $3.5 billion by 2024.
The FDA also recently granted priority review to Ultomiris, a monoclonal antibody and long-acting C5 inhibitor that works by preventing hemolysis, as a new treatment for another rare disease: hemolytic uremic syndrome. The agency said it plans to make a final decision about that indication in October. In an effort to continue expanding the drug’s reach, Alexion is also initiating phase 3 trials for at least two other rare diseases and is exploring the use of Ultomiris to treat amyotrophic lateral sclerosis (ALS) and primary progressive multiple sclerosis.
AbbVie’s up-and-coming upadacitinib hasn’t won FDA approval just yet, but the agency is expected to make a decision in the third quarter of this year. And if the treatment gets the go-ahead for the key therapeutic areas AbbVie is targeting — rheumatoid arthritis (RA), Crohn’s disease, ulcerative colitis, psoriatic arthritis and atopic dermatitis — the market potential will be huge. Several studies have also shown that the drug, a JAK inhibitor that blocks a pathway linked to inflammation, has out-performed the world’s best-selling drug, Humira, as a treatment for RA.
EvaluatePharma predicts that sales for upadacitinib will reach $2.2 billion by 2024.
AbbVie is also on the verge of launching another anti-inflammatory hit with risankizumab (Skyrizi), which is being investigated to treat several of the same indications as upadacitinib. So far, clinical trial results for Skyrizi have been positive, with one study showing that the drug cleared a significant number of patients of psoriasis symptoms even after the treatment was stopped.
The FDA approved Skyrizi, an IL-23 inhibitor, for patients with plaque psoriasis in April. EvaluatePharma predicts that sales for the drug will reach $2.08 billion by 2024, and AbbVie estimates that its peak sales will reach $4-5 billion.
Several gene therapies topped EvaluatePharma’s list, with Bluebird Bio’s Zenteglo leading the pack. In March, the European Medicines Agency granted Bluebird a conditional marketing approval for Zenteglo, which was developed to treat the underlying genetic cause of beta thalassemia, paving the way for a full approval later this year. The FDA is expected to make its final decision early next year.
Meanwhile, Bluebird is also investigating the use of Zenteglo for sickle cell disease, an indication that would help propel the treatment to blockbuster levels. But like other gene therapies, the price for Zenteglo is likely to be an issue. In the EU, Bluebird has set the price near $2 million, but the company says it is working with each country to create a reimbursement process that will allow access to patients who need it.
Despite the pricing controversy, EvaluatePharma predicts that Zenteglo will pull in about $1.9 billion in sales by 2024.
There’s been a lot of excitement over Aimmune Therapeutic’s AR101, the first-ever treatment for peanut allergies. Although approval for the drug, which was designed to combat dangerous allergic reactions in children who have been exposed to peanuts, was slowed down by the government shutdown in December, Aimmune says it is working with the agency to move the approval process along. The company expects that it will get the green light in the EU this year and a final word from the FDA by January 2020.
EvaluatePharma estimates that sales for AR101 will reach $1.8 billion within the next five years.