Top 5 Highly-Anticipated New Drugs Highlighted in Pipeline Report


As more novel therapies emerge from the pipeline and enter the market, it is crucial for pharmacists to monitor new drug approvals that could be lifechanging for their patients.

A surge of groundbreaking new drug developments, many of which touch the specialty space, have created excitement over the potential of treating and curing rare and life-threatening diseases. As more novel therapies emerge from the pipeline and enter the market, it is crucial for pharmacists to monitor new drug approvals that could be lifechanging for their patients.

OptumRx’s Drug Pipeline Insights Report highlights 5 influential drugs to watch for in the pipeline based on adoption, efficacy, and expected cost.

  • Onasemnogene abeparvovec (Zolgensma, AveXis)

Gene therapy onasemnogene abeparvovec, which was just approved by the FDA last week, is indicated for the treatment of patients less than 2 years of age with spinal muscular atrophy (SMA). The drug is an adeno-associated virus vector-based gene therapy that targets the cause of SMA, which is a leading genetic cause of infant mortality. The drug is administered as a 1-time intravenous infusion, whereas existing treatment nusinersen (Spinraza) involves injecting the drug into the spinal cord every 4 months.

Although onasemnogene abeparvovec has the potential to cure SMA, it has been priced at $2.125 million, or an annualized cost of $425,000 per year for 5 years, making it the most expensive medication to hit the market.

“However, when considering that Zolgensma is a 1-time treatment (compared to a lifelong regimen of recurring treatments), Zolgensma has the potential to reduce overall costs associated with SMA, and most patients will want to try it,” according to the report.

  • NKTR-181 (Nektar)

NKTR-181, a new abuse-deterrent opioid analgesic, has a novel molecular structure that differs from existing opioid analgesics. Due to its slow penetration into the brain in clinical trials, the drug shows the potential to attenuate the euphoric effect usually attributed to other pain medications. However, studies also found that patients had similar levels of drug liking compared with other opioids, sparking concern over the efficacy of its abuse deterrence properties in real world settings, according to the report.

If the anticipated benefits are proven, the adoption rates of NKTR-181 could be high due to the current need for safer alternatives to opioid medications. The New Drug Application (NDA) for NKTR-181 was filed in July 2018.

  • Golodirsen (Sarepta)

Golodirsen targets a small subpopulation of patients with Duchenne muscular dystrophy (DMD), a rare genetic disease cause by a mutation in the gene responsible for production of the muscle protein dystrophin. Golodirsen is engineered to treat patients with a genetic mutation subject to skipping exon 53 of the dystrophin gene, which is approximately 8% of patients with DMD.

According to the report, “this drug is notable because it provides a treatment option for a serious condition with no other treatment options available and if approved, the majority of eligible DMD patients will seek treatment with it.”

The FDA provided a regulatory action date of August 19, 2019, for golodirsen.

  • Upadacitinib (AbbVie)

AbbVie’s NDA for upadacitinib for the treatment of adult patients with moderate-to-severe rheumatoid arthritis (RA) was granted priority review by the FDA in February 2019. Upadacitinib is a once-daily oral Janus kinase (JAK) 1-selective inhibitor with a mechanism of action similar to other oral JAK inhibitors, such as tofacitinib and baricitinib. However, upadacitinib is especially notable because it is the only product in the class to demonstrate superiority versus adalimumab (Humira) in RA at its expected approved dose, which could lead to widespread adoption, according to the report.

Read more about data on upadacitinib here.

  • Tafamidis meglumine (Vyndaqel)

Tafamidis meglumine, a first-in-class oral treatment for transthyretin amyloid cardiomyopathy (ATTR-CM), was approved in early May 2019 for the condition. ATTR-CM is a rare and life-threatening disease, with approximately 100,000 individuals living with the condition in the United States, of whom just 1% to 2% are currently diagnosed. Prior to this approval, there were no available treatments for ATTR-CM, only symptom management and, in rare cases, heart transplant.

According to the report, the approval of this therapy is significant for several reasons. Not only is it the first and only treatment approved for ATTR-CM, but it offers the feasibility of being taken orally and has the potential to lower cardiovascular-related hospitalizations and mortality. The cost for tafamidis meglumine is high, however, at $250,000 per patient per year. High adoption could potentially create pressure on treatment affordability, the report concluded.


OptumRx. OptumRx Drug Pipeline Insights Report. Accessed May 29, 2019.

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