BioMarin Reports the US FDA’s sNDA Acceptance for Full Approval of Voxzogo in Achondroplasia

Shots: The US FDA has accepted an sNDA for Voxzogo (vosoritide) for full approval in children with achondroplasia (PDUFA: Feb. 28, 2027) The sNDA was supported by long-term safety & efficacy data from the ongoing 111-205, 111-208, & 111-302 studies, demonstrating clinically meaningful improvements in growth & key skeletal measures, incl. body proportionality & arm span…

ByRidhi RastogiJuly 14, 2026

TOP 20

Top 20 M&A of 2025 by Total Deal Value

Shots: 2025 pharma M&A was defined by fewer but more transformational deals, with companies prioritizing commercial-stage assets, late-stage pipelines, and platform technologies capable of reshaping long-term growth and strengthening core therapeutic franchises The year’s momentum was led by three standout transactions, J&J’s $14.6B acquisition of Intra-Cellular Therapies, Novartis’ $12B deal for Avidity Biosciences, and Merck’s…

ByPrince GiriJanuary 30, 2026

NEWS

BioMarin to Acquire Amicus Therapeutics for ~$4.8B

Shots: BioMarin has entered into a definitive agreement to acquire Amicus, strengthening BioMarin's commercial portfolio with Galafold (migalastat) for Fabry Disease & Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for Pompe Disease As per the deal, BioMarin will acquire Amicus for $14.5/share in an all-cash transaction for a total equity value of ~$4.8B; closing is expected in Q2’26 Amicus also…

ByRidhi RastogiDecember 22, 2025

NEWS

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

INSIGHTS+

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

INSIGHTS+

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

INSIGHTS+

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

INSIGHTS+

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 sNDA Acceptance for Full Approval of Voxzogo in Achondroplasia

Top 20 M&A of 2025 by Total Deal Value

BioMarin to Acquire Amicus Therapeutics for ~$4.8B

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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