Pfizer’s Panzyga Receives FDA Approval for Rare Neurological Disease

On February 12th, Pfizer announced it had received FDA approval for Panzyga to treat chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disease.

CIDP is a rare neurological disease that affects around 40,000 in the US. It is characterized by gradual motor and sensory loss due to loss of myelin, a coating around the nerves that allow for electric signals to be transmitted correctly through the body. Current first-line treatments include corticosteroids, therapeutic plasma exchange, and immunoglobulins.

The most common treatment is intravenous immunoglobulins. To determine maintenance dose and frequency of treatment, it is recommended to titrate the medication to suit the patients’ needs. However, clinical trials normally examine one single dose concentration, so titrating the immunoglobulin could lead to patients receiving doses that are not effective or are not effective in large clinical trials. Medications that are tested and approved for multiple concentrations are necessary to meet the patient’s needs.

Panzyga

Panzyga was created by the Swiss company, Octapharma and subsequently licensed by Pfizer to market and commercialize in the US. Panzyga is a 10% human normal immunoglobulin intravenous solution previously approved to treat primary humoral immunodeficiency, chronic immune thrombocytopenia, and now approved for CIDP.

The approval was based on a prospective, double-blind, randomized, multi-center, Phase 3 clinical trial that included 142 patients in 9 countries. This clinical trial was the first of its kind to evaluate multiple maintenance dosing options. The standard treatment is first, a dose of 2.0 g/kg followed by a maintenance dose of 1.0 g/kg. This clinical trial evaluated the two additional maintenance doses, 0.5 g/kg and 2.0 g/kg, administered every three weeks for 6 months. The results showed that almost 80% of patients had a decrease in inflammatory neuropathy cause and treatment (INCAT) disability score with the 1.0g/kg dose. Also, there was a dose-dependent improvement in the other two groups, with 64.7% of patients improving in the 0.5 g/kg group and 91.7% of patients improving in the 2.0 g/kg group.

Panzyga was well tolerated, with the most common adverse effects being headache, fever, dermatitis, and blood pressure increase.

“Each patient with CIDP has different treatment needs, and we have found that having one approved dosing option is not always optimal,” said Angela Lukin, Global President, Hospital Business Unit, Pfizer Inc. “The approval of this new indication with additional dosing options helps address an unmet patient need by providing healthcare providers with the ability to choose an approved dose that’s right for patients.”

Competitors on the Horizon

A new class of drugs that is emerging as a treatment for CIDP is antibody therapies. Currently, there at least three Phase 2 clinical trials looking at the safety and efficacy of different antibody treatments. These include Efgartigimod, developed by Argenx, which is an antibody fragment that targets the Fc receptor. Rozanolixizumab from UCB Biopharma, which blocks the interaction of FcRn and IgG. The final one is Rituximab, which binds to the CD20 receptor. The University of Kansas Medical Center is sponsoring the clinical trial for Rituximab. If any of these therapies prove to be effective, it could reduce the market share of Panzyga.

Related Article: G1 Therapeutics’ Drug Reduces Chemo Related Side Effects, Nabs FDA OK

References

1. https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-panzygar-treatment-adults-chronic
2. https://rarediseases.org/rare-diseases/chronic-inflammatory-demyelinating-polyneuropathy/
3. https://www.octapharma.com/news/press-release/2020/final-results-from-the-procid-study/
4. https://clinicaltrials.gov/ct2/show/NCT03861481
5. https://clinicaltrials.gov/ct2/show/NCT04281472
6. https://clinicaltrials.gov/ct2/show/NCT04480450

Author

Daniel Ojeda