BioMarin Reports the US FDA’s sBLA Acceptance & Priority Review of Palynziq (Pegvaliase-pqpz) to Treat Phenylketonuria

Shots: The US FDA has accepted sBLA & granted priority review to Palynziq for the treatment of adolescents (12-17yrs.) with phenylketonuria (PDUFA: Feb 28, 2026) sBLA was supported by the P-III (PEGASUS) trial comparing Palynziq to diet alone in 55 adolescents with phenylketonuria, which showed reduced blood phenylalanine (Phe); results were presented at ICIEM’25 Palynziq…

ByRidhi RastogiOctober 31, 2025

INSIGHTS+

Insights+: The US FDA New Drug Approvals in July 2023

Shots: The US FDA approved 5 NDAs and 2 BLA in July 2023, leading to treatments for patients and advances in the healthcare industry. The CDER and CBER approved 76 novel products in 2023 In July 2023, the major highlights drugs were Beyfortus (nirsevimab) approval for the prevention of RSV lower respiratory tract disease in…

BySenior EditorAugust 16, 2023

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Insights+: The US FDA New Drug Approvals in June 2023

Shots: The US FDA approved 12 NDAs and 3 BLA in June 2023, leading to treatments for patients and advances in the healthcare industry. The CDER and CBER approved 69 novel products in 2023 In June 2023, the major highlights drugs were Talzenna + Xtandi approval for prostate cancer, Rystiggo for generalized myasthenia gravis PharmaShots…

BySenior EditorJuly 19, 2023

TOP 20

Top 20 Cell and Gene Therapy Companies Based on 2022 Total Revenue

Shots: Standing tall and gradually paving its way through extensive research, Cell & Gene Therapy is a beacon of hope when treating rare and inherited diseases. Cell and gene therapy aims to prevent, treat, and potentially reduce the effects of the underlying cause of genetic diseases. Propelled by constant innovatory winds, the cell and gene…

ByPrince GiriMay 23, 2023

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Insights+: EMA Marketing Authorization of New Drugs in August 2022

The EMA approved 5 New Chemical Entity (NCE) and 4 Biologic Drugs in August 2022, leading to treatments for patients and advances in the healthcare industry In August 2022, the major highlights drugs were Lynparza’s approval for breast cancer, Imbruvica (ibrutinib) for chronic lymphocytic leukaemia, Sunlenca (lenacapavir) for HIV Infection PharmaShots has compiled a list…

BySenior EditorSeptember 28, 2022

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Insights+: The US FDA New Drug Approvals in November 2021

The US FDA has approved 6 NDAs and 1 BLAs in 2021, leading to treatments for patients and advances in the health care industry The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 68 novel products in 2021 Additionally, last year in 2020, the US FDA…

BySenior EditorDecember 16, 2021

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PharmaShots’ Key Highlights of Second Quarter 2021

The US FDA has approved 6 NDAs and 2 BLAs in 2021, leading to treatments for patients and advances in the health care industry The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 37 novel products in 2021 Additionally, last year in 2020, the US FDA…

BySenior EditorJuly 7, 2021

BioMarin Reports the US FDA’s sBLA Acceptance & Priority Review of Palynziq (Pegvaliase-pqpz) to Treat Phenylketonuria

Insights+: The US FDA New Drug Approvals in July 2023

Insights+: The US FDA New Drug Approvals in June 2023

Top 20 Cell and Gene Therapy Companies Based on 2022 Total Revenue

Insights+: EMA Marketing Authorization of New Drugs in August 2022

Insights+: The US FDA New Drug Approvals in November 2021

PharmaShots’ Key Highlights of Second Quarter 2021

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