These are the top 3 drugs recently approved — or soon to be approved by FDA — to watch in 2020.
While there are numerous drugs in the pipeline, pharmacy benefit manager OptumRx singled out three top medications expected to have the most impact on payers on patients in the second half of 2020.
In the PBM’s Pipeline Insights Report: 3 Drugs to Watch, OptumRx highlights treatments for breast cancer, spinal muscular atrophy, and Duchenne muscular dystrophy. Here are the top 3 drugs to watch in the second half of this year:
1. Sacituzumab govitecan-hziy (Trodelvy, Immunomedics) was approved in late April to treat patients with metastatic triple-negative breast cancer (TNBC) who have received at least two prior therapies for metastatic disease.
TNBC is an aggressive breast cancer that accounts for up to 20% of all breast cancer cases.
Trodelvy is a novel antibody-drug conjugate (ADC), which combines both biologically engineered antibodies andtraditional chemotherapy drugs, OptumRx said. It is administered intravenously on a weekly basis over a 21-day cycle.
“Trodelvy is the first ADC targeted therapy for TNBC. There is a high unmet need for treatments in this subtype of breast cancer because of the size of the overall breast cancer population and the aggressive nature of TNBC,” OptumRx said.
In fact, analysts estimate that Trodelvy could reach $1.44 billion in global sales in 2024 global sales, initially as a third-line treatment for late-stage TNBC. The drug is also being evaluated in earlier settings and other cancers.
2. The investigational drug risdiplam (F. Hoffmann-La Roche, PTC Therapeutics, SMA Foundation) would be the first oral therapy to treat spinal muscular atrophy (SMA).
SMA is a rare group of severe neuromuscular disorders that leads to progressive muscle weakness and atrophy. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies, OptumRx said.
“There is an unmet need for treatments for SMA, and especially for an all-oral option. There are currently only two FDA- approved drugs for SMA,” OptumRx said.
Nusinersen (Spinraza, Biogen) requires repeated, invasive injections into the cerebrospinal fluid in the spinal cord. The other alternative isonasemnogene abeparvovec (Zolgensma, AveXis) which is a one-time IV-infused gene therapy.
FDA is expected to approve the medication on or before August 24, 2020, according to OptumRx.
“While Spinraza does require invasive spinal injections, it has been widely used in SMA patients since its market entry. Practically speaking, the competitive advantage for risdiplam will rest mainly in its oral route of administration, and perhaps, a lower cost,” OptumRx said.
3. Viltolarsen (Nippon Shinyaku) is in development to treat specific forms of Duchenne muscular dystrophy (DMD). DMD is a rare genetic disorder that affects young boys.
There is a significant unmet need for treatments for DMD since it is associated with substantial sickness and death,” OptumRx said.
If approved, viltolarsen would be the third exon-skipping drug for DMD, and the second specifically for the 8% of patients with a specific mutation.
Golodirsen (Vyondys 53TM, Sarepta Therapeutics) is also approved for the mutation, but there is limited efficacy data and unknown clinical benefit with its use, OptumRx said.
The FDA is expected to make a decision on whether or not to approve viltolarsen in the third quarter of this year.
Read more: FDA clears novel breast cancer drug