One of the metrics used to gauge the industry's progress is the number of new medicines that receive regulatory approval annually. The returns from the first quarter indicate that the innovation engine is in good shape and biopharma companies appear to be on pace to match the totals they achieved in the past couple of years. However, they have a tough act to follow as far as new molecular entities (NMEs) are concerned, since last year 46 new medicines got the green light, a total that ranks as the second highest number of approvals all time just behind the 1996 total when 53 new medicines were approved. (See BioWorld Insight, Jan. 8, 2018.)

During the first quarter of this year six NMEs (four NDAs and two BLAs) were approved for marketing by the FDA, which is in line with the six approved in the same period of 2016, but just half of the 12 that were approved last year. There were 10 NMEs approved by the end of 2015's first quarter. With the industry track record running at an average of 32 NMEs annually over the past decade, it looks as though it is on track to reach the same number this year. (See New Molecular Entities approved, below.)

NMEs targeting cancer

Among the six NMEs that have reached market so far, two are indicated for cancer. Janssen Research & Development LLC, a unit of Johnson & Johnson (J&J), received approval for apalutamide, well ahead of its targeted PDUFA date, to treat patients with non-metastatic castration-resistant prostate cancer (nmCRPC), and whose disease has ceased to respond to medical or surgical treatments that lower testosterone but has yet to spread.

The decision was based on data showing that the drug, which will be marketed as Erleada, decreased the risk of distant metastasis or death by 72 percent vs. placebo while extending median metastasis-free survival by 24.3 months in nmCRPC patients. Erleada blocks the effects of androgen on the tumor. (See BioWorld, Feb 15, 2018.)

According to J.P. Morgan research, the drug could achieve annual sales of $1.2 billion by 2022.

People suffering from a rare group of cancers that affect the pancreas or gastrointestinal tract – known as gastroenteropancreatic neuroendocrine tumors (GEP-NETs) – are faced with limited treatment options after initial therapy fails to keep the cancer from growing, explained Richard Pazdur, director of the FDA's Oncology Center of Excellence. That is why the FDA approval of Lutathera (lutetium Lu 177 dotatate) indicated for adult patients with somatostatin receptor-positive GEP-NETs will be welcomed. Pazdur also noted that this is the first time a radiopharmaceutical, has been approved for the treatment of these tumors.

The drug was developed by Advanced Accelerator Applications SA, but is now under the banner of Novartis AG, which acquired the company.

New HIV treatments

Canadian company Theratechnologies Inc., of Montreal, and its partner, Taimed Biologics Inc., of Taiwan, China, received approval for Trogarzo (ibalizumab-uiyk), the first HIV treatment to hit the market with a new mechanism of action in more than a decade. The CD4-directed post-attachment HIV-1 inhibitor, for use in combination with other antiretroviral treatments (ARTs) is designed to treat HIV-1 infection in adults with multidrug-resistant HIV-1 infection who failed an existing ART regimen.

Mackie Research Capital Corp. estimates that there are about 8,000 to 10,000 multidrug-resistant HIV patients in the U.S. who are intolerant to current antiretroviral treatments. With Trogarzo priced at a wholesale acquisition cost (WAC) of $118,000/year, and Theratechnologies expecting the net selling price to be at a 30 percent discount to that price, 2020 sales of the drug could reach over $400 million. (See BioWorld, March 8, 2018.)

Gilead Sciences Inc. also added to its arsenal of HIV treatments when the FDA approved Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg, or BIC/FTC/TAF) for use in HIV-1-infected adults who are either treatment-naïve or have been virologically suppressed on a stable antiretroviral regimen for a minimum of three months.

In a research note, RBC Capital Markets analyst Brian Abrahams said, "Biktarvy is being positioned by GILD as the optimal therapy for all treatment-naive HIV [patients], which we believe will enable it to take substantial share over time."

Consensus estimates for annual sales of the once-daily medicine by 2021 is well above $4 billion.

Cystic fibrosis

Vertex Pharmaceuticals Inc. gained the FDA green light for its cystic fibrosis (CF) drug, Symdeko (tezacaftor/ivacaftor and ivacaftor). It is indicated to treat CF in people ages 12 and older with two copies of the F508del mutation in the CF transmembrane conductance regulator gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor.

The new treatment becomes the company's third CF medicine and offers an important option for many patients, including those eligible who may be interested in a different treatment. (See BioWorld, Feb. 14, 2018.)

At the end of the first quarter, Ilumya (tildrakizumab-asmn) to treat adults with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy became the sixth NME to be approved. Mumbai, India-based Sun Pharmaceutical Industries Ltd. licensed global rights to the drug from a subsidiary of Merck & Co. Inc. in 2014. (See BioWorld, March 28, 2018.)

By the numbers

Including the six NMEs, the FDA handed out 40 drug approvals (three ANDAs, two BLAs, 12 NDAs, 10 sBLAs and 13 sNDAs) in the first quarter, compared with 34 by the end of the first quarter of 2017 and 42 at the end of the first quarter of 2016.

Thousand Oaks, Calif.-based Amgen Inc.'s supplemental BLA application for Blincyto (blinatumomab) to treat minimal residual disease-positive B-cell precursor acute lymphoblastic leukemia – an indication that has no approved treatments – received the only accelerated approval awarded in the period. Eight drug approvals had priority review; four had orphan designation; and seven took the 505(b)(2) pathway.

In terms of indications, topping the list were 12 targeting cancer, seven infectious diseases therapies, five for neurology/psychiatric, and three for dermatologic conditions.