The FDA has granted orphan drug status to Gilead Sciences Inc’s experimental drug remdesivir, providing several incentives and benefits for the company’s research.1 The drug has also been given Expanded Access, or “compassionate use” to address the pandemic.2

Orphan drug designation is awarded to investigational drugs for rare diseases or conditions, which are defined by the FDA as conditions with fewer than 200,000 patients in the United States.1  

While remdesivir is not FDA-approved to treat the novel coronavirus (COVID-19), the orphan drug status would provide 7 years of exclusive marketing in the United States and tax credits on research and development costs once the drug was approved.1

Remdesivir has been a key player in COVID-19 research, having demonstrated in vitro and in vivo activity in animal models against Middle East Respiratory Syndrome (MERS) and Severe Acute Respiratory Syndrome (SARS), which are also coronaviruses. The drug is a nucleotide analog with broad-spectrum antiviral activity.3

Gilead has initiated 2 phase 3 clinical trials to investigate the drug’s effects on patients with COVID-19. According to the company’s website, the studies began enrolling patients in March of 2020 and will enroll approximately 1000 patients in the initial phase, in countries with high COVID-19 prevalence.4

The first study will evaluate the safety and efficacy of 5-day and 10-day dosing durations or remdesivir for patients with severe cases of COVID-19. The second study will evaluate the same dosing regimens for patients with moderate COVID-19, compared to standard of care alone.4

According to the Associated Press, some experts are saying the FDA’s approval is a misuse of orphan drug designation.1 While it offers incentives for Gilead, experts say the 7-year exclusivity would limit other companies from developing drugs for COVID-19 at a time when more research and development is much needed. Additionally, while there are still just about 50,000 cases in the US, many more are expected to be diagnosed, which could potentially overtake the 200,000 limit for orphan drug designation.1

Following the compassionate use approval from the FDA, Gilead announced that the company would no longer be accepting individual compassionate use requests due to high demand, with the exception of requests for pregnant women and children under 18 years old with severe confirmed COVID-19.5

“Gilead is mapping out options to make access to investigational remdesivir more widely available through expedited regulatory processes should it demonstrate the potential to be a safe and effective treatment option based on the results of preliminary clinical trials,” Gilead said in a statement.6


REFERENCES
  1. Perrone M, Lardner R. Potential coronavirus treatment granted rare disease status. Associated Press; March 25, 2020. https://apnews.com/608d4bb9d76bb1077dec5409a7e2459e. Accessed March 25, 2020.
  2. Coppock K. FDA Announces Two Drugs Given ‘Compassionate Use’ Status in Treating COVID-19. Pharmacy Times; March 19, 2020. https://www.pharmacytimes.com/news/fda-announces-two-drugs-approved-for-compassionate-use-in-treating-covid-19. Accessed March 25, 2020.
  3. Gilead Sciences Inc. About Remdesivir. https://www.gilead.com/purpose/advancing-global-health/covid-19/about-remdesivir. Accessed March 25, 2020.
  4. Gilead Sciences Inc. Remdesivir Clinical Trials. https://www.gilead.com/purpose/advancing-global-health/covid-19/remdesivir-clinical-trials. Accessed March 25, 2020.
  5. Gilead Sciences Inc. Emergency Access to Remdesivir Outside of Clinical Trials. https://www.gilead.com/purpose/advancing-global-health/covid-19/emergency-access-to-remdesivir-outside-of-clinical-trials. Accessed March 25, 2020.
  6. Gilead Sciences Update On The Company’s Ongoing Response to COVID-19 [news release]. Updated March 22, 2020. https://www.gilead.com/purpose/advancing-global-health/covid-19. Accessed March 25, 2020.