5 highlights of FDA drug approval in the second quarter of 2021

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A few days ago, the foreign biopharmaceutical website BiopharmaDive released 5 highlights that deserve attention in the US FDA drug approval in the second quarter of 2021.

1, aducanumab

Despite extensive research, Alzheimer's disease (AD) remains one of the most challenging areas in drug development.

But treatment options for AD will soon change.

However, approval is far from certain.

However, industry analysts have not yet determined that aducanumab will be sentenced to death.

When the regulator recently decided to extend the review of aducanumab, some analysts saw this as a sign that the regulator was combing through more data to support the approval.

2.

For many years, the main treatment for Pompe disease has been long-term treatment with Lumizyme, an enzyme replacement therapy.

However, Sanofi has yet to prove this hypothesis.

Nevertheless, the approval of Sanofi avalglucosidase alfa may raise the threshold of other treatments being developed, including an oral drug from Amicus Therapeutics, and some gene therapies developed by Roche, Bayer, and Avrobio.

3.

Positive results from early studies in the UK and elsewhere cannot confirm that the vaccine is effective in elderly people.

In late March, AstraZeneca reported in a phase 3 clinical study conducted in the United States that this vaccine is safe and very effective in preventing COVID-19, laying the foundation for the FDA's review.

The advisory board meeting held this month may reflect these twists and turns.

4.
20vPnC

20vPnC is a 20-valent pneumococcal vaccine developed by Pfizer.
It is currently undergoing priority review by the US FDA.
The target date of action is June 2021.
The vaccine is used in adults aged 18 and over to prevent invasive diseases and pneumonia caused by the serotype of Streptococcus pneumoniae in the vaccine.
Previously, the FDA has granted the vaccine breakthrough drug designation.

In addition to the 13 serotypes in the existing standard vaccine product Prevnar 13 (Prevnar 13), 20vPnC also covers 7 serotypes related to high mortality, antibiotic resistance and/or meningitis.
Prevnar 13 is Pfizer’s overweight pneumococcal vaccine.
With global sales of US$5.
85 billion in 2020, it is Pfizer’s best-selling product, but the product will lose patent protection in 2026.

If the new vaccine 20vPnC is approved for marketing, it will help consolidate Pfizer’s franchise rights.
The FDA will review the application of 20vPn for adults aged 18 and over in June this year.
This is a relatively small group compared to Prevnar 13, which can be used for children and infants as young as 6 weeks.
.
But Pfizer's goal is to eventually apply it to young people.
The company has released phase 2 clinical data for 20vPnC in infants and children.

At present, Merck’s 15-valent pneumonia vaccine V114 is also undergoing priority review by the US FDA.
The vaccine is used to prevent invasive pneumococcal disease in adults aged 18 and above.
The target action date is July 18, 2021.
The design of V114 follows the company's older 23-valent vaccine, Pneumovax 23 (Neumovax).
Like Pfizer's new vaccine, V114 can be used to prevent some key serotypes that cause invasive pneumococcal disease, and these key serotypes have not been included in the vaccine products already on the market.

5.
pimavanserin

Pimavanserin is a key asset of Acadia Pharmaceuticals and the active pharmaceutical ingredient of its only marketed product Nuplazid.
The product was approved for marketing in April 2016 and became the first drug to treat psychiatric symptoms such as hallucinations and delusions experienced by Parkinson’s disease patients.
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As the first drug that selectively targets the 5-HT2A receptor, pimavanserin's unique pharmacology has created a new drug category—selective serotonin inverse agonist (SSIA), which not only preferentially targets 5-HT2A The HT2A receptor can also avoid the side effects of dopamine receptor and other receptor activation that most psychiatric drugs have.

In 2020, Nuplazid's sales have increased by 30%.
In recent years, Acadia has been trying to promote further growth of Nuplazid by expanding its therapeutic indications.
Last year, the company submitted a Supplemental New Drug Application (sNDA) for Nuplazid to the US FDA for the treatment of hallucinations and delusions associated with dementia-related psychosis (DRP), with a target action date of April 3, 2021.

According to the Acadia announcement, the FDA has issued a complete response letter (CRL) to the aforementioned sNDA.
This indicates that the agency has completed the review and determined that the sNDA cannot be approved in its current form.
Although the company previously reached an agreement with the FDA Psychiatric Products Department on the design of the key Phase 3 HARMONY study, the broad DRP patient population was analyzed as a single group.
However, the CRL pointed out that certain subgroups of dementia lack statistical significance, and certain less common subtypes of dementia have insufficient numbers of patients and lack sufficient efficacy data to support approval.

The HARMONY study reached the pre-specified primary and secondary endpoints: in the treatment of DRP-related hallucinations and delusions, strong and convincing data confirmed the clinical and statistical superiority of Nuplazid over placebo, which is An agreed prerequisite for DRP indications.
The statistical separation according to the minimum number of dementia subgroups and specific patients is not within the pre-specified requirements.

Acadia Company expressed a strong protest against the FDA's decision.
The company said that during the entire review process, the agency did not raise any questions about the agreed research agreement, including the issues raised in the CRL.
We will immediately request a Class A meeting to work with the FDA to resolve the CRL problem and determine a fast way to approve the application of Nuplazid in the treatment of DRP.

Reference source: 5 FDA approval decisions to watch in the second quarter