Christine Blank

While approval of several drugs have been delayed due to COVID-19, FDA has still cleared more than 30 new drugs this year — ahead of its average rate, a new OptumRx report says.

Here are the top 5 drugs in the pipeline for 2020 — either approved recently or expected to garner FDA clearance this year — according to OptumRx’s most recent Drug Pipeline Insight Report.

 Valoctocogene roxaparvovec (Roctavian, BioMarin) to treat hemophilia A in adults. While an FDA decision on whether or not the approve the medication was expected by August 21, the agency requested additional data from BioMarin.

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“Existing treatments for this condition are extremely expensive, costing up to $500,000 per year. Valoctocogene roxaparvovec would offer a one-time gene therapy that could replace the need for these treatments in some patients,” OptumRx wrote.

The projected wholesale acquisition cost (WAC) for valoctocogene roxaparvovec is expected to be in the range of $2 million to $3 million per one-time dose.

 Filgotinib (Jyseleca, Gilead Sciences) to treat moderate-to-severe rheumatoid arthritis (RA). The drug has yet to obtain FDA approval.

“Just one day prior the scheduled decision date, the FDA announced that it will require additional data from two ongoing safety studies of the drug. Relatedly, the FDA has expressed concerns regarding the overall benefit/risk profile of filgotinib at the 200 mg. dose used in trials,” OptumRx wrote. “Industry analysts speculate that if these concerns cannot be satisfied, the entire development program may be dropped by the manufacturer.”

 Brexucabtagene autoleucel (Tecartus, Kite, a Gilead company) treats adult patients with relapsed or refractory mantle cell lymphoma (MCL), a rare and aggressive form of non-Hodgkin’s lymphoma (NHL). It was approved in late July.

Notably, Tecartus is a chimeric antigen receptor T-cell (CAR T) therapy, which leverages and strengthens the power of a patient’s immune system to attack cancer cells.

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“Tecartus becomes the first CAR T-cell therapy for MCL, offering an additional treatment option for patients with relapsed or refractory MCL that only requires a one-time dose administered by IV infusion,” OptumRx wrote.

In trials with a median follow-up period of 12.3 months, 93% of patients responded to a single infusion of Tecartus, including 67% of patients who achieved a complete response.

 Subcutaneous ofatumumab (Kesimpta, Novartis), approved in late August, treats relapsing forms of multiple sclerosis (MS). Ofatumumab is currently available in intravenous (IV) form as Arzerra, which is FDA approved for the treatment of chronic lymphocytic leukemia.

Kesimpta will be entering a crowded MS marketplace, competing not only with Aubagio, but also with other well established oral and injectable products with different mechanisms of action, OptumRx said.

“Analysts say that being self-administered by subcutaneous injection would give Kesimpta a key advantage,” OptumRx said.

 Tafasitamab-cxix (Monjuvi, MorphoSys AG), approved in late July, is the first FDA-approved, second-line regimen for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

Monjuvi is to be used in combination with lenalidomide (Revlimid). It will be competing with polatuzumab vedotin (Polivy), which was approved in June 2019 for a similar indication.

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Some of the top medications were approved this summer, while others are awaiting FDA approval.

Top 5 drugs in the pipeline

FDA granted Breakthrough Therapy Designation to Toripalimab (Junshi Biosciences) to treat nasopharyngeal carcinoma. The drug is the first anti-PD-1 antibody from China to receive BTD, according to Junshi.

“The Breakthrough Therapy designation granted by FDA recognizes the significant clinical benefits Toripalimab has shown for the treatment of nasopharyngeal carcinoma,” said Ning Li, MD, CEO of Junshi Biosciences, in a 

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“Junshi prioritizes cancer types with high prevalence in China such as nasopharyngeal carcinoma, lung and liver cancer, but we found that great unmet medical needs also exist in other countries where patients are endangered by this deadly disease,” Li said. “The BTD will allow us to work with the FDA closely to bring the therapy to patients worldwide expeditiously.”

However, Junshi did not say when the drug would be available in the U.S.

The anti-PD-1 monoclonal antibody received its first approval for second-line treatment of metastatic melanoma in China in 2018.

In March, FDA granted Toripalimab in combination with axitinib in the treatment of mucosal melanoma orphan drug designation. And in May, the treatment was granted orphan drug designation by FDA to treat nasopharyngeal carcinoma.

The company has carried out more than 30 clinical studies covering more than 10 indications for the treatment.

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The treatment is the first anti-PD-1 antibody from China to receive Breakthrough therapy designation.

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 the first 5-minute COVID-19 test that allows results to be read directly from a testing card, the White House said it would work with Abbott Laboratories to make 150 million tests available.

 for the first antigen test with that design, which is similar design to some pregnancy tests. The test, BinaxNow COVID-19 Ag Card, is fast and efficient for healthcare providers and patients and does not need the use of an analyzer, FDA said in a press release.

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“This means people will know if they have the virus in almost real-time. Due to its simpler design and the large number of tests the company anticipates making in the coming months, this new antigen test is an important advancement in our fight against the pandemic,” said Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health.

On August 27, the White House announced the $760 million deal with Abbott to produce 150 million tests, The Washington Post 

 that it would ship “tens of millions of tests in September, ramping production to 50 million tests a month in October.”

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“This is a major development that will help our country to remain open, get Americans back to work, and kids back to school!” White House Press Secretary Kayleigh McEnany tweeted.

However, the government did not say where the tests will be distributed.

Here’s how the test works: a healthcare provider swabs the patient’s nose and twirls that sample on a test card with a testing reagent added. After waiting 15 minutes, the provider reads the results directly from the testing card. One line indicates a negative result; two lines indicate a positive result.

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The pharma maker will initially produce 150 million of the 5-minute tests.

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FDA approved the first-cell-based gene therapy to treat mantle cell lymphoma (MCL).

Brexucabtagene autoleucel (Tecartus, Kite, a Gilead company), a chimeric antigen receptor (CAR) T cell therapy, received Priority Review and Breakthrough Therapy Designation by FDA.

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“This approval is yet another example of customized treatments that use a patient’s own immune system to help fight cancer, while using a scientific advance in this promising new area of medicine,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in a 

. “We’re seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.”

Each dose of Tecartus is a customized treatment created using a patient’s own immune system to help fight the lymphoma, FDA said. The patient’s T cells are collected and genetically modified to include a new gene that facilitates the targeting and killing of the lymphoma cells. The modified T cells are then infused back into the patient.

“Despite promising advances, there are still major gaps in treatment for patients with MCL who progress following initial therapy,” said Michael Wang, MD, lead investigator of the Tecartus trial and professor at The University of Texas MD Anderson Cancer Center, in a Kite 

. “Many patients have high-risk disease and are more likely to keep progressing, even after subsequent treatments.

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“The availability of Tecartus as the first-ever cell therapy for patients with relapsed/refractory MCL provides an important option with a response rate of nearly 90% and early clinical evidence suggesting durable remissions in later lines of therapy,” Wang added.

The safety and efficacy of Tecartus was established in a multicenter clinical trial of 60 adults with refractory or relapsed MCL who were followed for at least 6 months after their first objective disease response. The complete remission rate after treatment with Tecartus was 62% and the treatment boasted an objective response rate of 87%.

Tecartus’s label carries a boxed warning for cytokine release syndrome (CRS), a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and for neurologic toxicities. Both CRS and neurologic toxicities can be fatal or life-threatening.

Because of the risk of CRS and neurological toxicities, Tecartus is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU).

FDA is also requiring Kite to conduct a post-marketing observational study involving patients treated with Tecartus.

The chimeric antigen receptor (CAR) T cell therapy is the first of its kind for mantle cell lymphoma (MCL).

Author | Christine Blank

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