Thankful for Biopharma Breakthroughs

Despite the constant need for social distancing, mask-wearing, and the isolation and economic uncertainty that resulted from the outbreak, there is still much to be thankful for when families gather around a virtual table to break bread and carve the turkey this year.

For so many, 2020 has been a bleak year filled with uncertainty and anxiety directly related to the COVID-19 pandemic that has surged across the globe and led to the deaths of more than 1.4 million people, including close to 260,000 in the United States.

Despite the constant need for social distancing, mask-wearing, and the isolation and economic uncertainty that resulted from the outbreak, there is still much to be thankful for when families gather around a virtual table to break bread and carve the turkey this year. And one uniting bit of thankfulness the global community can share in is the prowess of the international pharmaceutical industry displayed to address COVID-19. Following the outbreak that originated in China, then spread across Asia and into Europe, the pharmaceutical industry pivoted on a dime to tackle the global threat. Ongoing research was put on the backburner and scientists began to focus on understanding the virus and assessing what medications could be used against it. The virus was also sequenced and hundreds of vaccine projects were initiated. The industry, along with scientists from various government agencies and academic institutions joined together in a united front against the global pandemic.

And those efforts are now beginning to pay off. In Russia and China, vaccines are already being distributed to front-line workers and manufacturing is ramping up for broader distribution. In the west, we are just weeks away from seeing the first coronavirus vaccine receive Emergency Use Authorization. The mRNA vaccine candidate developed by Pfizer and Germany-based BioNTech demonstrated 95% efficacy in clinical trials. The U.S. Food and Drug Administration (FDA) will review the data on Dec. 10.

When that medication is greenlit (as it most likely will be), the limited number of vaccines currently available will roll out within 24 hours and inoculation will begin. Fortunately, more vaccines will likely see approval in the United States and Europe, which means more people will receive some protection against the virus. Moderna reported vaccine efficacy of 94.5% and earlier this week, AstraZeneca also announced 90% efficacy from its vaccine candidate. Novavax and Johnson & Johnson are expected to release data soon, as will Merck and other companies.

The vaccine approvals are the proverbial light at the end of the tunnel that is COVID-19. High rates of inoculation will lead to herd immunity against the virus and that is something for which to be thankful.

But, it’s not just vaccines that have been developed for COVID-19. The FDA recently approved two antibody treatments for the virus, Eli Lilly’s bamlanivimab and Regeneron’s REGN-COV2, which had previously been used to treat the COVID-19 diagnosis of President Donald Trump. Both of the antibody treatments do have limits for their use. They are not meant for COVID-19 patients who require supplemental oxygen or are on ventilators.

Gilead Sciences remdesivir broke through as the first COVID-19 drug to receive full approval from the FDA as a medication that can shorten the time of infection for infected patients. Despite its approval, Remdesivir has received a rocky reception, with the World Health Organization recommending against its use due to limited capabilities. Other drugs have also received similar receptions over the course of the pandemic. While remdesivir has clinical data supporting its approval, other medications such as hydroxychloroquine have only anecdotal data backing up any efficacy against the virus. Still, those COVID-19 patients who have benefited from the treatments are surely thankful for any edge against the virus they received.

COVID-19 has certainly dominated our landscape over the past nine months, but other illnesses continue to negatively impact the human condition. COVID research has been a primary focus, but that has not put a halt to the development of treatments for other diseases, including rare diseases.

This week, Alnylam won approval for Oxlumo (lumasiran), the first drug approved by the FDA for primary hyperoxaluria type 1, an ultra-rare genetic disease that causes deposits of calcium oxalate crystals to form in the kidneys and urinary tract, which can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure and system organ dysfunction. Also this week, the FDA approved Eiger PharmaceuticalsZokinvy, the first drug approved to treat Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies. The two genetic diseases cause premature, rapid aging that dramatically decreases the lifespan of children affected. In June, Novartis became the first company to the finish line with a treatment for Adult-Onset Still’s Disease (AOSD), a rare auto-inflammatory disease of unknown origin. Ilaris (canakinumab), was previously approved for Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged 2 years and older.

These approvals and others not mentioned that improve quality of life and stave off premature death are all things the pharmaceutical industry and its countless, dedicated employees have provided for which we should be thankful.