In the Pipeline: Specialty Drugs to Watch in 2021

Despite the pandemic, manufacturers are continuing to develop new drugs, especially specialty medications. Read on for the most important specialty drugs you should be paying attention to in 2021.

Last year, the FDA approved over 50 new medications. As in previous years, new approvals continue to skew towards specialty drugs for treating small patient populations. With difficulties enrolling patients into clinical trials during the global pandemic, the years ahead may see fewer new drugs. However 2021 looks to be another busy year for drug releases and reviews.

Our pharmacists continually monitor the drug development pipeline, paying special attention to the products closest to approval. This allows us to help clients stay ahead of the curve on new and upcoming drugs. Here are the specialty medications to watch in 2021:

Oncology

2020 was an important year for oncology drug development, with cancer treatments making up over a third of new medication approvals. In addition, many existing cancer drugs were approved for additional indications. Early signs suggest this trend will continue in 2021, with many more medications scheduled for FDA review.

Paclitaxel is a common intravenous chemotherapy medication. The FDA will be reviewing a new formulation for metastatic breast cancer that allows for oral use in the first quarter of 2021. With promising trial results, this version may replace intravenous paxclitaxel in different cancers in the future.

Orgovyx™ (relugolix) was approved as an oral androgen deprivation therapy (ADT) to treat advanced prostate cancer at the end of 2020. Prostate cancer is the second most common form of cancer in men and ADT is a first-line treatment for cancer that cannot be treated with surgery or radiation. Until the approval of relugolix, ADT could only be accomplished using injectable medications or castration.

Tagrisso® (osimertinib) was approved in late 2020 for adjuvant treatment in certain patients with non-small cell lung cancer. While osimertinib and similar medications are used to treat more advanced stages, this is the first time these drugs have been approved for use in preventing disease recurrence following surgery, something traditionally accomplished with chemotherapy.

Keytruda® (pembrolizumab) is already approved for treating 16 forms of cancer and Opdivo® (nivolumab) is already approved for treating 10 forms of cancer. Both are likely to see additional indications in 2021. Pembrolizumab is in review for breast and gastroesophageal cancer treatment, while nivolumab was just approved for kidney cancer on January 22, 2021.

Anemia in patients with chronic kidney disease

For patients with chronic kidney disease, anemia is a significant factor in needing blood transfusions. To reduce the need for transfusions, many patients use injectable erythropoiesis-stimulating agents. Multiple manufacturers are developing hypoxia-inducible factor prolyl hydroxylase (HIF-PF) inhibitors, a new class of oral medication. All of the following products are undergoing phase III trials including roxadustat, vadadustat, daprodustat and molidustat. Of these drugs, roxadustat is likley to be the first drug approved in this class.

Atopic dermatitis

Atopic dermatitis, or eczema, is a condition characterized by itchy skin. In more severe cases, this itchiness can effect quality of life, including disrupted sleep, broken skin from scratching and increased risk of infections. A number of products are in development for this condition, especially for its more severe forms.

Dupixent® (dupilumab) is an injectable interleukin antagonist used to treat moderate to severe atopic dermatitis. Previously approved for patients 12+ years, it was approved for 6-11 year olds in May 2020. It is being studied in patients as young as 6 months old, with trial results expected in 2022. Dupilumab was recently approved for severe asthma and nasal polyps, and is being studied for other respiratory and skin conditions.

Tralokinumab, lebrikizumab and nemolizumab are all injectable interleukin antagonists either in clinical trials or under review.

Oral JAK inhibitors are another option for treating atopic dermatitis. They are expected to work more quickly than injectable interleukin antagonists, but come with serious safety concerns. These include abrocitinib, Rinvoq™ (upadacitinib), Olumiant® (baricitinib) and Jakafi® (ruxolitinib).

Type 1 diabetes

Type 1 diabetes is a chronic condition that destroys beta cells, which produce insulin in the pancreas. Teplizumab will be reviewed by the FDA in the first half of 2021. If approved, it would be the first drug that can help slow the development of type 1 diabetes. While the standard treatment is to replace insulin, teplizumab helps preserve the body’s beta cells, potentially preventing the development of diabetes. Because it works to prevent beta cell loss, teplizumab will likely only impact undiagnosed or newly diagnosed patients.

Other areas of interest

For each drug that makes it to market, many more fail in clinical trials. NASH and Alzheimer’s disease are two conditions that continue to generate significant demand, but haven’t had any successful FDA approvals.

Non-alcoholic steatohepatitis (NASH) is the most severe form of non-alcoholic fatty liver disease (NAFLD) and is associated with fat build-up on the liver, inflammation and liver fibrosis. Severe forms of the disease are a leading cause of liver cirrhosis and transplantation, and is linked with obesity, pre-diabetes and diabetes. Ocaliva® (obeticholic acid) was anticipated to be approved as the first NASH drug in 2020, but the FDA found the evidence to be insufficient to grant accelerated approval. Five additional Phase III drug candidates may have topline trial results in 2021 or 2022.

Alzheimer’s disease has had no new therapeutic products approved by the FDA in over a decade, with several promising clinical candidates failing in phase III trials. One recent reported failure, aducanumab, was brought back into development after a post-hoc data analysis combined from two separate trials showed more favorable results than the original trials. An FDA decision is anticipated by March 2021.

As with last year, 2021 is shaping up to be significant year for specialty drug releases and development. As the year unfolds, our pharmacists will continue to monitor new developments to help your organization stay ahead of the curve.