Top 4 drugs in the pipeline

Dutta

In OptumRx’s recent “Drug Pipeline Insight Report,” the pharmacy benefit manager reveals the top drugs in development for the second quarter of 2019.

“OptumRx actively monitors the drug development pipeline based on a wide range of professional resources, as well as our ongoing insight into pharmaceutical companies’ drug development programs. That data is shared with our Pharmacy & Therapeutics Committee so that they can consider drugs before they come to market and informs our clinically-based formulary design process,” wrote Sumit Dutta, chief medical officer for OptumRx, in the report.

The 4 drugs that OptumRx expects to make the biggest impact in terms of their clinical quality, effectiveness and cost, include:

• NKTR-181 (Nektar Therapeutics) is a new opioid analgesic that differs from existing opioid analgesics in that it has a molecular structure that makes it more difficult to abuse, with a lower likelihood of developing dependency, according to OptumRx. “NKTR-181 is particularly notable given the ongoing opioid epidemic in the US. In clinical trials, the chemical structure caused a delayed penetration into the brain with the potential for less euphoria usually attributed to other pain medications,” the PBM wrote.

Related: Top 3 New Drug Approvals

However, studies also showed that patients had similar levels of “drug liking” compared to other opioids, thus leading some to be skeptical that NKTR-181’s benefits will translate into less abuse in clinical practice. “While NKTR181 may provide hope for a safer opioid, if the abuse deterrence properties do not translate into reality, the drug will not have superiority over existing options, many of which are available as generics. If the anticipated benefits are proven, then adoption rates could be high since patients and prescribers will view it as a safer alternative to other opioids,” OptumRx wrote.

• Upadacitinib (ABT-494, AbbVie) is a JAK1 selective inhibitor to treat rheumatoid arthritis, Crohn's disease, ulcerative colitis, atopic dermatitis, psoriatic arthritis, and other inflammatory conditions. “Upadacitinib is notable because it is the only product in the class to demonstrate superiority vs. Humira (adalimumab) in RA patients at its expected approved dose. This data could give it an edge over competitors, potentially leading to widespread adoption,” OptumRx wrote.

RA is a large drug category, accounting for 25% of all specialty drug spending. Olumiant and Xeljanz have a price of $26,000 and $54,500 per year, respectively, the PBM noted.

However, class-associated safety remains a potential concern, especially increased risk of serious infection, liver toxicity, and the potential for malignancies.

    3. Golodirsen (SRP-4053, Sarepta) treats Duchenne muscular dystrophy (DMD), a rare genetic disease caused by a mutation in the gene responsible for production of the muscle protein dystrophin. “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,” OptumRx wrote.

Related: FDA Clears Novel Diabetes, Tropical Disease Therapies

Golodirsen targets a small subpopulation of DMD patients (8%) and addresses the genetic defect in a unique manner called “exon skipping”, according to OptumRx. “The drug essentially provides a bridge over the genetic mutation found on exon 53 thus enabling production of a shorter but functional form of the muscle protein dystrophin,” the PBM wrote.

• Tafamidis meglumine (Vyndaqel, Pfizer), approved by FDA in May, is a first-in-class oral treatment for the rare disease transthyretin amyloid cardiomyopathy (ATTR-CM). With this condition, proteins accumulate in the heart, leading to heart failure, heart block, and sudden death.

Vyndaqel is the first and only FDA-approved treatment for ATTR-CM, according to OptumRx. “Other existing medications treat the associated symptoms, and, in rare cases, heart (or heart and liver) transplants are an option,” the PBM wrote.

Another benefit of Vyndaqel is that it can be taken as an oral capsule. The other available drugs to treat the condition require either subcutaneous injections weekly or intravenous infusions every 3 weeks, according to OptumRx.

Read more: Top 6 Cancer Drug Trends