2020 inventory: peak sales forecast of new drugs approved by FDA and EMA in the United States! Gilead, vertex, Roche, Pfizer, Novartis

2020 is a "eventful year" for the pharmaceutical industry. Many pharmaceutical companies around the world are developing covid-19 therapeutic drugs or vaccines at a fanatical pace. So far, covid-19 has claimed about 1.94 million lives worldwide.

With the implementation of the blockade in various countries and the suspension of on-site inspection by drug regulatory agencies in many countries, it was originally expected that the approval rate of new drugs would be much lower by mid-2020. But in July last year, after studying the new drug approvals of the US Food and Drug Administration (FDA) and the European Drug Administration (EMA), it was found that the US FDA had approved 33 new drugs by the end of June. This figure is within the average range of the past two years, giving pharmaceutical companies hope in the impact of the covid-19 pandemic.

As of December 31, FDA has granted 58 new drug approvals, 53 chemical preparations have been approved by the center for drug evaluation and research, and 5 biological preparations have been approved by the center for biological evaluation and research. This is even higher than the number of new drug approvals in 2019 (54). Among them, Lilly and Gilead each have four new drug indications approved, tied for the first. According to the treatment field, 23 items were approved in tumor field, 16 items in infectious diseases and infections, 8 items in genetic diseases, 8 items in neurology, 3 items in immunology, and 2 items in gastrointestinal diseases.

Source: Pharma compass


EMA is also very busy this year. They have issued 75 positive approval opinions. Among the 75 approved new drug indications, Novartis won 8 and was in the leading position, followed by Pfizer and Sanofi with 4 each.

According to the treatment field, 21 items were approved in tumor field, 19 items in infectious diseases and infections, 6 items in genetic diseases, 5 items in immunology, 5 items in lung / respiratory diseases, 4 items in heart / vascular diseases, 4 items in endocrine diseases and 4 items in neurological diseases.

Source: Pharma compass

Year marked by EUA

As the covid-19 pandemic rages around the world, drug emergency use authorization (EUAs) has made headlines. In 2020, FDA issued 10 EUAs, among which Pfizer / biontech and Moderna's covid-19 vaccine are the most representative authorized drugs.

There are also controversial products in EUAs. For example, in March this year, the US FDA released the EUA for the treatment of covid-19 with oral chloroquine phosphate and hydroxychloroquine sulfate. By June, however, the EUA had been withdrawn because the FDA found that chloroquine and hydroxychloroquine were unlikely to be effective in the treatment of covid-19.

At the same time, EUAs also produced heavy drugs. In May, the US FDA approved the emergency use of the Gilead antiviral drug remdesivir. In the following months, the drug rapidly climbed to the top of the list of best-selling drugs in 2020. In October last year, the drug became the first drug approved by the US FDA to treat covid-19 patients who need hospitalization. Although the prospect of the drug in covid-19 inpatients remains uncertain, especially in the World Health Organization (who) - led trial, which showed little effect on 28 day mortality or length of hospital stay in covid-19 patients, analysts still expect a peak sales of $3.5 billion.

Top 10 sales forecast of FDA approved drugs in 2020

Source: Pharma compass

Drugs approved by EMA in 2020

Top 10 sales forecast

Source: Pharma compass

Gillide's $21 billion acquisition of immunomedicine

In addition to the success of redaway, there are many events worth summarizing in the global pharmaceutical industry in 2020, including Gilead. In April, the antibody drug conjugate (ADC) trodelvy (sacituzumab govitecan hziy) of immunomedicine was approved by FDA for use in adult patients with metastatic triple negative breast cancer who had received at least two treatments before. These tumors account for 15% - 20% of breast cancer patients. Therefore, trodelvy was selected into the list of heavyweight drugs with the most sales potential approved by FDA in 2020. Currently, it is predicted that trodelvy's sales will reach US $2.151 billion by 2026.

However, just five months later, in September 2020, Gilead decided to take a major move to acquire immunomedicine, a biotechnology company, for $21 billion. The deal, completed in October, strengthens Gilead's cancer portfolio and adds a potential blockbuster drug to it.

Immunomedicine is planning to submit a supplementary biological agent license application (BLA) to support trodelvy's full approval in the United States in the first quarter of this year. In addition, immunomedicine is expected to apply for regulatory approval for trodelvy in Europe in the first half of 2021. At the same time, ongoing studies are also evaluating the potential of the drug in the treatment of non-small cell lung cancer and other types of solid tumors.

Risdiplam approved by Roche and PTC therapeutics

Following the approval of troveldy by FDA, Roche and PTC's drug evrysdi (risdiplam) have been approved as the first oral drug for the treatment of spinal muscular atrophy (SMA). Until four years ago, there was no treatment available for this rare genetic disease. In 2016, the drug spinraza (nusinersen) developed by Bojian and Ionis was approved, becoming the world's first drug for the treatment of SMA. The RNA based drug is administered intrathecally and needs to be infused at the doctor's office three times a year. In May 2019, Novartis gene therapy zolgensma (onasemgene abeparvovec) was approved, becoming the first gene therapy for SMA in the world. The drug was given by single and single intravenous infusion.

Evrysdi's approval offers patients and their families another unique option: daily oral medication at home. Roche is priced according to the weight of patients, with a maximum cost of $340000 per year, which is far lower than the listed competitive products of Bojian and Novartis.

Vertex's kaftrio is approved by EMA

Trikafta is a combination of ivacaftor, tezacaftor and elexacaftor. Its excellent clinical data enabled FDA to approve trikafta within three months after vertex submitted its marketing application and five months before FDA took action.

In the new drug sales forecast approved by FDA in 2019, trikafta ranks first, and its sales volume is expected to reach 3.935 billion US dollars by 2024. In June 2020, the EMA human medicine committee (CHMP) adopted a positive opinion and proposed to grant the marketing authorization of vertex combination drugs and conduct marketing under the trade name of kaftrio. After opening the European market, the drug will further create peak sales.

Many setbacks in the approval of new therapies

2020 is not going well. In fact, there have been many twists and turns in this year - 44 drugs have not been approved by the US FDA. Bristol Myers Squibb (BMS) is one of the companies that suffered setbacks. As part of the $74 billion acquisition of celgene, BMS offered shareholders contingent rights (CVR) shares. According to the agreement, CVR will enable new base shareholders to obtain a one-time expected earnings of $9 per share, provided that the three former new base drugs are approved within a predetermined period of time.

In March last year, the US FDA approved ozanimod, a sphingosine 1-phosphate receptor agonist produced by BMS and Bluebird bio for the treatment of recurrent multiple sclerosis, which was greatly ahead of the deadline of December 31, 2020. But there is still no progress in the approval of the remaining two drugs.

In May, the two companies announced that they had received a letter of refusal from the US FDA to apply for a biological agent license (BLA) for their car-t therapy idecabtagene vicleucel (IDE CEL) in patients with untreated, severe, recurrent and refractory multiple myeloma. After the initial review, FDA considered that the chemical, manufacturing and control (CMC) module of IDE cel needs to provide further details to complete the review. The approval decision shall be given before March 31, 2021.

Subsequently, BMS announced that the US FDA extended the BLA validity of another CD19 oriented car-t therapy, lisocabtagene maraleucel (LISO CEL), by three months to November 16, 2020. LISO cel is used in adult patients with relapsed or refractory large B-cell lymphoma who have received at least two previous treatments. However, on November 16, FDA continued to inform LISO cel that the BLA review could not be completed. There is still no news.

In addition, the U.S. FDA also refused to approve Novartis's lipid-lowering drug inclisiran. In 2019, Novartis acquired the medicines company for us $9.7 billion, because Novartis is very interested in the innisiran owned by the latter. The drug has been approved for sale by the European Commission.

Novartis said the FDA had no concerns about the efficacy or safety of inclisiran. The rejection was due to unresolved conditions related to the inspection of pharmaceutical facilities. And the FDA didn't conduct an on-site inspection.

Summary: a year of blocking and seizing time

Like pharmaceutical companies, global drug regulators are working frantically to speed up drug approval and EUAs, especially for covid-19 treatments and vaccines.

In addition to covid-19, cell and gene therapy has been more widely adopted and approved in this year, and some innovative drugs have been approved, such as relugolix (the first oral gonadotropin-releasing hormone receptor antagonist for the treatment of adult patients with advanced prostate cancer), perotralstat (the first oral once daily plasma kallikrein inhibitor for the prevention of age 12 and advanced prostate cancer) More than 50% of patients with hereditary angioedema in adults and children), lumasiran (a hao1 targeted small interfering RNA used to treat primary hyperoxaluria type 1 in children and adults and reduce urinary oxalate levels), and osilodostat (for adult Cushing's disease).

In addition, there are some interesting "non covid" medical breakthroughs during 2020. One of the breakthroughs is the development of a single tablet that combines four drugs that lower blood pressure and cholesterol, as well as aspirin, which reduces the risk of heart disease.

Despite the impressive agility shown by pharmaceutical companies and regulators, and the fact that many countries have awarded the EUA to covid-19 vaccine, the ongoing pandemic has indeed put humans in a dilemma for months. The novel coronavirus crisis will not be the last pandemic. If we do not solve climate change and animal problems, efforts to improve human health will be doomed to failure. Therefore, from this pandemic, the pharmaceutical industry needs to learn and summarize many things.

reference material:

1. New Drugs Approvals by FDA and EMA: 2020 Recap

2. AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy (SMA)

3. Bristol Myers' $9 Celgene CVR is teetering on the brink of disaster as the FDA delays the liso-cel application yet again

4. CVR tied to BMS's Celgene takeover dealt blow as FDA again delays liso-cel review