The U.S. FDA has approved 48 drugs in 2019¹, several of which represent advanced, first-in-class therapies with billion dollar blockbuster potential. The following are some drug approvals expected to have a major impact, both therapeutically and financially.
 
In August, AbbVie received approval from the FDA for RINVOQ (upadacitinib), a once-daily oral Janus kinase (JAK) inhibitor, for the treatment of adults with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response or intolerance to methotrexate (MTX-IR). The approval was supported by data from one of the largest registrational Phase III programs in RA with approximately 4,400 patients evaluated across all treatment arms in five studies. Analysts predict that Rinvoq could generate as much as $2.2 billion in annual revenue over the next four years. 
 
Additionally, earlier this year AbbVie received FDA approval for Skyrizi (risankizumab-rzaa), for the treatment of plaque psoriasis, which is expected to recoup some of the revenue losses associated with Humira competition. EvaluatePharma projects that Skyrizi will generate annual sales of $3.2 billion by 2024.
 
In October 2019, Novartis received approval for Beovu from the FDA for the treatment of wet AMD. Beovu (brolucizumab) is the most clinically advanced humanized single-chain antibody fragment (scFv). Single-chain antibody fragments are highly sought after in drug development due to their small size, enhanced tissue penetration, rapid clearance from systemic circulation and drug delivery characteristics. EvaluatePharma estimates that the drug could make nearly $1.2 billion annually by 2024.
 
AveXis, a Novartis company, received approval from FDA for Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients with spinal muscular atrophy (SMA) with SMN1 gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis. EvaluatePharma estimates the market potential for the gene therapy will reach $1.6 billion by 2024.

In April, The Janssen Pharmaceutical Companies of Johnson & Johnson received accelerated approval from the FDA for BALVERSA (erdafitinib) for the treatment of adults with locally advanced or metastatic bladder cancer, which has susceptible fibroblast growth factor receptor (FGFR)3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy. BALVERSA is the first FGFR kinase inhibitor approved by the FDA. EvaluatePharma estimates that Balversa bring in nearly $1.2 billion by 2024.

In November, Celgene and Acceleron Pharma received approval from the FDA for REBLOZYL (luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. REBLOZYL is the first and only FDA-approved erythroid maturation agent, representing a new class of therapy which works by regulating late-stage red blood cell maturation to help patients reduce their RBC transfusion burden. According to Jefferies analysts, the drug could reach $2 billion in yearly sales. 
 
In May, Pfizer received approval from the FDA for both VYNDAQEL (tafamidis meglumine) and VYNDAMAX (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM. 
 
Transthyretin amyloid cardiomyopathy is a rare, life-threatening disease characterized by the buildup of abnormal deposits of misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure. Previously, there were no medicines approved to treat ATTR-CM. EvaluatePharma expects Vyndaqel will achieve $2.1 billion in revenue by 2024.
 
In March, Novartis received FDA approval for Mayzent (siponimod), the first oral drug to treat secondary progressive multiple sclerosis (SPMS) with active disease. SPMS is a debilitating form of multiple sclerosis (MS) characterized by progressive and irreversible neurological disability. EvaluatePharma projects that Mayzent could generate $1.3 billion in revenue annually by 2024.

1. Novel drug approvals for 2019