What to Watch in 2020: Specialty Drugs

It’s the New Year and that means there’s a new batch of drugs in the pipeline. Here’s what you should be paying attention to in 2020.

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If you read last week’s article on drug development, you will know that it is a lengthy process that takes years of research and testing. Today, we will be focusing on specialty drugs that are approaching the end of Phase III clinical trials or that are being reviewed by the FDA in 2020. In some cases, we will also highlight medications in an earlier testing stage that show great promise. Just know that these won’t be coming to market this year, or possibly even next year.

With that in mind, here are the specialty drugs to watch in 2020:

SICKLE CELL DISEASE

Sickle cell disease is a serious hereditary disorder involving misshaped red blood cells which results in serious complications such as severe pain, infections, acute chest syndrome, stroke and premature death. This disease affects over 100,000 Americans and is especially prevalent in Black, Hispanic, Middle Eastern, Mediterranean and Indian populations. Unfortunately, few effective treatment options have been available.

Two treatments launched in early 2020, including Oxbryta™ (voxelotor) oral tablets and Adakveo® (crizanlizumab), an intravenously infused product.
While they are still years from potential approval, LentiGlobin and BCL11A shRNA (miR) are two gene therapy treatments to watch. Currently undergoing early clinical trials (Phase I/II), these products may have the potential to significantly impact the treatment landscape.

DUCHENNE MUSCULAR DYSTROPHY

Duchenne muscular dystrophy (DMD) is a severe, hereditary type of muscular dystrophy that affects 400 to 600 boys in the United States annually. While the mainstay of treatment for most patients has been corticosteroids, more targeted disease-modifying therapies are being developed and approved.

The FDA approved Vyondys 53™ (golodirsen) in December 2019, the first new treatment for DMD since Exondys 51® (eteplirsen) was launched in 2016. Both drugs are mutation-specific, and therefore only addresses small sections of the population with DMD, ranging from 8% to 13% by mutation.
Casimersin, developed by the same manufacturer as Exondys 51 and Vyondys 53, targets the exon 45 mutation and is expected to file for approval shortly.
Viltolarsen, the first product from a competitive manufacturer in this space, will compete with Vyondys 53 in targeting exon 53 mutations. This product is currently undergoing FDA review.

CYSTIC FIBROSIS

Cystic fibrosis is a hereditary condition that damages the lungs and digestive system. Although significant treatment breakthroughs have occurred in recent years, many patients have mutations that lack effective treatment. With a new treatment option, that may change.

Trikafta™ (elexacaftor/ivacaftor/tezacaftor), a breakthrough therapy for cystic fibrosis, was approved by the FDA in late 2019. Unlike previous therapies, it is approved for 90% of the population with cystic fibrosis. This includes 12,000 patients eligible for treatment with existing therapies and 6,000 patients with a previously untreatable mutation.¹ Trikafta is currently approved for patients 12 years and older, although a study of patients 6 to 12 years of age is expected to conclude early this year with a potential indication for this population in 2021.

NON-ALCOHOLIC STEATOHEPATITIS (NASH)

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 obesity, pre-diabetes and diabetes. We anticipate the first NASH drug may be approved in 2020, with another filing in 2020 and five more Phase III drug candidates that may pursue approval in 2021 and 2022.

Ocaliva® (obethicholic acid) is currently approved for primary biliary cholangitis, a rare but serious autoimmune disease of the liver. Obeticholic acid was the first product with a successful Phase III trial for NASH and is currently under review by the FDA for that indication.
Elafibranor is a dual PPARα & -δ agonist that targets fat deposition and inflammation, the earlier components of NASH, rather than later-stage fibrosis. The drug also appears to have the benefit of decreasing low-density lipoproteins (LDL), otherwise known as “bad” cholesterol. Interim Phase III data for elafibranor is expected soon and, depending on results and filing timeline, may receive FDA approval in 2021.

SEIZURE DISORDERS

A variety of seizure disorders exist, with epilepsy being the most well-known. The following upcoming medications are meant to address some of the less common seizure disorders.

Fintepla® (fenfluramine) is being investigated as an add-on to existing therapies for Dravet syndrome, a rare and serious form of epilepsy. After positive Phase III results, this product was filed with the FDA for review in September 2019 with a decision expected in March 2020.
Xcopri (cenobamate) is an oral drug that is added on to existing therapies for the treatment of partial seizures. Cenobamate received FDA approval for treating adults in late 2019 but is not yet available.²
Ganaxolone is another oral drug designed to treat rare genetic seizure disorders, including CDLK5 deficiency and PCDH19-related epilepsy. Phase III data for ganaxolone is expected in 2020, with a potential FDA filing in late 2020.

SPINAL MUSCULAR ATROPHY

Spinal muscular atrophy (SMA) is a genetic disorder that results in inadequate levels of SMA protein, which leads to wasting and weakness in skeletal muscles. The most severe form of the disease, SMA Type I, typically results in a life expectancy of less than 2 years. Less severe forms are more common, with a wide variety of severity largely correlated with the age of symptom onset.

Risdiplam is an oral drug that helps to sustain sufficient SMA protein levels that has been studied in all forms of SMA, including patients previously treated with Spinraza (nusinersen), Zolgensma (onasemnogene abeparvovec) and other investigational therapies. Based on promising early trial data, the FDA has granted risdiplam priority review, with a decision expected by May 24, 2020.³
Zolgensma (onasemnogene abeparvovec) was launched in May 2019 and is priced at $2.125 million for a one-time treatment. This gene therapy replaces the SMN1 gene for patients less than two years old, allowing the production of sufficient SMA protein.

SPECIALTY GENERICS

Several new or upcoming specialty generics may have significant impact in 2020. Availability of generic products may be dependent upon patent expirations, FDA approval, results of ongoing patent litigation and specific terms of confidential settlement agreements. Some key examples of products that could potentially become available include:

Everolimus is the generic of Afinitor®, an antineoplastic chemotherapy drug. While most strengths of this product became available in December, the most utilized strength, 10 mg, is expected later this year.
Teriparatide is the generic of Forteo®, an osteoporosis treatment.
Dimethyl fumarate is the generic of Tecfidera®, which is used to treat multiple sclerosis.
Pomalidomide is the generic of Pomalyst®, which is used to treat multiple myeloma.
Emtricitabine/tenofovir disoproxil fumarate is the generic of Truvada, which is used to treat and prevent HIV.
Efavirenz/emtricitabine/tenofovir disoproxil fumarate is the generic of Atripla, used to treat HIV.

Overall, 2020 is shaping up to be an exciting year for specialty drug releases. Be sure to join us again next week when we will explore the pipeline of the most important non-specialty medications for 2020.

FDA approves new breakthrough therapy for cystic fibrosis. FDA.gov. https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis. Published October 21, 2019. Accessed January 3, 2020.
FDA approves new treatment for adults with partial-onset seizures. FDA.gov. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-adults-partial-onset-seizures. Published November 21, 2019. Accessed January 3, 2019.
FDA grants priority review to Roche’s risdiplam for spinal muscular atrophy. Roche. https://www.roche.com/media/releases/med-cor-2019-11-25.htm. Published November 25, 2019. Accessed January 13, 2020.