Attention Biotech Investors: Mark Your Calendar For January PDUFA Dates

Year-end reviews by the Food and Drug Administration produced mostly positive outcomes, with most therapies passing the regulatory hurdle without any hiccups.

Despite the problems posed by the pandemic for the conduct of clinical trials and regulatory inspections of facilities, the biopharma sector continued to innovate at a brisk pace this year. New molecular entity approvals, considered a measure of innovation, totaled 53 in 2020 compared to 48 in 2019.

Notable among the NME approvals for December were BioCryst Pharmaceuticals, Inc.'s BCRX heredity angioedema treatment Orladeyo, MacroGenics Inc's MGNX Margenza for treating breast cancer and Myovant Sciences Ltd's MYOV Orgovyx to treat prostate cancer.

The FDA also accorded emergency use authorizations for the investigational vaccines of Pfizer Inc. PFE-BioNTech SE – ADR BNTX and Moderna Inc MRNA.

Here are the key PDUFA goal dates for the unfolding month:

Merck Seeks Approval For Heart Failure Drug

Company: Merck & Co., Inc. MRK
Type of Application: NDA
Candidate: vericiguat
Indication: heart failure
Date: Jan. 20

Vericiguat is an orally administered soluble guanylate cyclase stimulator that is being evaluated, in combination with existing heart failure therapies, as a treatment option to reduce the risk of cardiovascular death and heart failure hospitalization in patients with symptomatic chronic heart failure with reduced ejection fraction, or HFrEF.

HFrEF occurs due to the inability of heart to eject blood sufficiently during the contraction phase. About 6.5 million people in the U.S. have heart failure, and approximately 40-50% of these patients have HFrEF, Merck said.

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Aurinia's Kidney Inflammation Drug Awaits Nod

Company: Aurinia Pharmaceuticals Inc AUPH
Type of Application: NDA
Candidate: voclosporin
Indication: lupus nephritis
Date: Jan. 22

Voclosporin is Aurinia's investigational drug, which is being evaluated for multiple indications. The FDA accepted the company's new drug application for voclosporin as a treatment option for lupus nephritis on July 21.

Lupus nephritis is serious inflammation of the kidneys caused by the autoimmune disease systemic lupus erythematosus.

In mid-December, Aurinia announced a licensing agreement with Otsuka Pharma to commercialize voclosporin in Europe and Japan.

Can Pfizer's Cancer Treatment Get Label Expansion?

Company: Pfizer Inc. PFE
Type of Application: sNDA
Candidate: Xalkori (crizotinib)
Indication: pediatric lymphoma
Date: Jan. 23

Xalkori is a tyrosine kinase inhibitor, which has already been approved by the FDA for the treatment of patients with metastatic non-small cell lung cancer, whose tumors are ALK-positive or ROS1-positive as detected by an FDA-approved test.

Pfizer has now filed to get approval for the drug to treat pediatric patients with relapsed or refractory systemic anaplastic large cell lymphoma that is ALK-positive.

Fourth Approval In The Cards For Amgen's Nplate

Company: Amgen, Inc. AMGN
Type of Application: sBLA
Candidate: Nplate
Indication: Hematopoietic Syndrome of Acute Radiation Syndrome.
Date: Jan. 28

Nplate, a peptibody protein, works by raising and sustaining platelet counts. It was initially approved by the FDA in 2008 for the treatment of thrombocytopenia in adults with chronic immune thrombocytopenic purpura.

Chronic ITP is a serious autoimmune disorder characterized by low platelet counts in the blood, which can lead to serious bleeding events.

Subsequently, in Dec. 2018, it was approved to treat pediatric patients, one year of age and older, with ITP. It received another label expansion in Oct. 2019, indicating it can be used to in newly diagnosed and persistent adult ITP patients who have had an insufficient response to corticosteroids.

The company is now seeking approval for Nplate as a treatment option for Hematopoietic Syndrome of Acute Radiation Syndrome.

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