Pharmaceutical Technology

SEPTEMBER 30, 2022

Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

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PharmaShots

APRIL 7, 2023

TG Therapeutics Receives EMA’s CHMP Positive Opinion of Briumvi (ublituximab-xiiy) for the Treatment of Relapsing Forms of Multiple Sclerosis Date: Apr 3, 2023 | Tags: TG Therapeutics, Briumvi, Ublituximab-xiiy, Multiple Sclerosis, Regulatory, EMA, CHMP BMS Receives EMA’s CHMP Positive Opinion of Breyanzi (lisocabtagene maraleucel) for (..)

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PharmaShots

MAY 12, 2023

Sanofi Reports P-IIIb Trial (HARMONIE) Results of Nirsevimab for the Prevention of Hospitalizations due to RSV-Related LRTD Date: May 12, 2023 | Tags: Sanofi, Nirsevimab, RSV-Related LRTD, Clinical Trial, P-IIIb, HARMONIE Trial G1 Therapeutics Presents Preliminary Results from P-II Trial of Trilaciclib for Triple-Negative Breast Cancer at ESMO 2023 (..)

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pharmaphorum

AUGUST 25, 2022

Johnson & Johnson has its first worldwide regulatory approval – in the EU – for Tecvayli, one of two much-anticipated bispecific antibodies the drugmaker has developed for patients with multiple myeloma. Tecvayli previously scored a priority medicines (PRiME) designation from the EMA and a breakthrough-therapy tag from the FDA.

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pharmaphorum

JULY 25, 2022

J&J filed for approval of the bispecific in the US in December based on the results of the phase 1b MajesTEC-1 study in myeloma patients who had been treated with a median of five lines of prior therapy. Most patients diagnosed with myeloma will relapse over the course of their disease, so the disease is still considered incurable.

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pharmaphorum

DECEMBER 17, 2021

Aduhelm (aducanumab) was approved by the FDA in June, but has been held back by a glacial rollout as clinicians have questioned the data on which the decision to clear the drug was based, plus a $56,000 per year price tag, which has in turn led to pushback by payers.

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European Pharmaceutical Review

MAY 11, 2023

Notably, data from LT-001, a 15-year ongoing observational study following the Phase I START patients, showed that children treated after presenting symptoms of SMA maintained all previously achieved milestones up to 7.5 1 This is assuming a $1M price tag for all gene therapies. years post-dosing.

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FDA 71