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Pipeline Monitor
Approval of Novartis CAR-T drug 'a new frontier'
Posted 1 September 2017
The FDA has green lighted Novartis' Kymriah (tisagenlecleucel) in a regulatory first which FDA Commissioner Scott Gottlieb has called "a new frontier in medical innovation".
Kymriah was OK'd for the treatment of patients up to 25 years old with B-cell precursor acute lymphoblastic leukaemia, making it the first cell-based gene therapy to be approved in any jurisdiction.
In the FDA's statement on the drug's approval, Gottlieb himself said the new class of drugs were an "inflection point in our ability to treat and even cure many intractable illnesses" as the CAR-T therapies use a patient's own individual T-cells genetically modified and infused back into the patient to kill cancer cells.
Approval for the drug came one month early for Novartis, who submitted Kymriah to the regulatory body in March winning a priority review and a unanimous vote from an FDA panel.
Kymriah's entry into the market will heat things up for the CAR-T space, which earlier this week saw Gilead make a USD12 billion acquisition of Kite Pharma's own CAR-T candidate, axicabtagene ciloleucel. Novartis has priced its new therapy at USD475 000, placing it into the lower bracket of expected prices and possibly putting pressure on Gilead to do the same.
The drug was approved based on one multicentre clinical trial which showed the drug had an overall remission rate of 83 per cent within three months among 63 young patients with the blood cancer.
And Roche will take care of side-effects
Riding on the success of Novartis' CAR-T approval, Roche's Actemra (tocilizumab) has won its seventh indication in the US as a treatment for cytokine release syndrome (CRS) induced by the new class of therapies.
Actemra was approved by the FDA to treat CRS in patients two years and older who have received CAR-T treatment. CRS is caused by an overactive immune response to the therapy and can be potentially severe and life-threatening.
The approval was based on a retrospective analysis of previous CAR-T clinical trials for blood cancers, which assessed the efficacy of Actemra in treating the CRS side-effect.
Roche won a priority review and orphan drug designation for the indication.
First Parkinsons dyskinesia drug approved
The FDA has approved Adamas' Gocovri (amantadine) for the treatment of dyskinesia in patients with Parkinson's disease also receiving levodopa-based therapy.
According to Adamas' company statement the drug is the first FDA-approved medicine for the treatment of dyskinesia in Parkinson's patients, which is characterised by unpredictable involuntary and non-rhythmic movements during waking hours.
Results from two phase 3 trials accompanied the submission showing the drug demonstrated statistically significant and clinically relevant reductions in dyskinesia in Parkinson's patients.
AstraZeneca scores first-line use for Faslodex
AZ's breast cancer drug Faslodex (fulvestrant) received FDA approval for the first-line treatment of metastatic or locally advanced breast cancer.
Already Ok'd for the indication in Europe, the drug saw its existing second-line indication expanded to now include first-line treatment of women with HR+ HER2- advanced breast cancer who have gone through menopause and have not received prior endocrine therapy.
Approval was based on a phase 3 clinical FALCON trial, which showed Faslodex increased median progression-free survival by around three months compared to patients receiving genericised aromatase inhibitor anastrozole.
Roche and Pfizer win speedy reviews
Roche picked up two priority reviews this week for drugs emicizumab and Gazyva (obintuzumab) from the FDA while Pfizer's drug Bosulif (bosutinib) also won a speedy review pathway.
Emicizumab was submitted to both the EU and US as a prophylaxis for haemophilia A while Gazyva, which is sponsored by Roche's Genentech arm, was filed as a first-line combination therapy with chemo to treat patients with follicular lymphoma, one of the most common blood cancers among adults.
Meanwhile Pfizer received a priority review from the regulatory body for Bosulif, indicated to treat Philadelphia chromosome positive chronic myeloid leukaemia.
Priority review pathways shorten approval review times to six months.
Yajun Ma
yajun.ma@lushmedia.com.au
Posted 1 September 2017
The FDA has green lighted Novartis' Kymriah (tisagenlecleucel) in a regulatory first which FDA Commissioner Scott Gottlieb has called "a new frontier in medical innovation".
Kymriah was OK'd for the treatment of patients up to 25 years old with B-cell precursor acute lymphoblastic leukaemia, making it the first cell-based gene therapy to be approved in any jurisdiction.
In the FDA's statement on the drug's approval, Gottlieb himself said the new class of drugs were an "inflection point in our ability to treat and even cure many intractable illnesses" as the CAR-T therapies use a patient's own individual T-cells genetically modified and infused back into the patient to kill cancer cells.
Approval for the drug came one month early for Novartis, who submitted Kymriah to the regulatory body in March winning a priority review and a unanimous vote from an FDA panel.
Kymriah's entry into the market will heat things up for the CAR-T space, which earlier this week saw Gilead make a USD12 billion acquisition of Kite Pharma's own CAR-T candidate, axicabtagene ciloleucel. Novartis has priced its new therapy at USD475 000, placing it into the lower bracket of expected prices and possibly putting pressure on Gilead to do the same.
The drug was approved based on one multicentre clinical trial which showed the drug had an overall remission rate of 83 per cent within three months among 63 young patients with the blood cancer.
And Roche will take care of side-effects
Riding on the success of Novartis' CAR-T approval, Roche's Actemra (tocilizumab) has won its seventh indication in the US as a treatment for cytokine release syndrome (CRS) induced by the new class of therapies.
Actemra was approved by the FDA to treat CRS in patients two years and older who have received CAR-T treatment. CRS is caused by an overactive immune response to the therapy and can be potentially severe and life-threatening.
The approval was based on a retrospective analysis of previous CAR-T clinical trials for blood cancers, which assessed the efficacy of Actemra in treating the CRS side-effect.
Roche won a priority review and orphan drug designation for the indication.
First Parkinsons dyskinesia drug approved
The FDA has approved Adamas' Gocovri (amantadine) for the treatment of dyskinesia in patients with Parkinson's disease also receiving levodopa-based therapy.
According to Adamas' company statement the drug is the first FDA-approved medicine for the treatment of dyskinesia in Parkinson's patients, which is characterised by unpredictable involuntary and non-rhythmic movements during waking hours.
Results from two phase 3 trials accompanied the submission showing the drug demonstrated statistically significant and clinically relevant reductions in dyskinesia in Parkinson's patients.
AstraZeneca scores first-line use for Faslodex
AZ's breast cancer drug Faslodex (fulvestrant) received FDA approval for the first-line treatment of metastatic or locally advanced breast cancer.
Already Ok'd for the indication in Europe, the drug saw its existing second-line indication expanded to now include first-line treatment of women with HR+ HER2- advanced breast cancer who have gone through menopause and have not received prior endocrine therapy.
Approval was based on a phase 3 clinical FALCON trial, which showed Faslodex increased median progression-free survival by around three months compared to patients receiving genericised aromatase inhibitor anastrozole.
Roche and Pfizer win speedy reviews
Roche picked up two priority reviews this week for drugs emicizumab and Gazyva (obintuzumab) from the FDA while Pfizer's drug Bosulif (bosutinib) also won a speedy review pathway.
Emicizumab was submitted to both the EU and US as a prophylaxis for haemophilia A while Gazyva, which is sponsored by Roche's Genentech arm, was filed as a first-line combination therapy with chemo to treat patients with follicular lymphoma, one of the most common blood cancers among adults.
Meanwhile Pfizer received a priority review from the regulatory body for Bosulif, indicated to treat Philadelphia chromosome positive chronic myeloid leukaemia.
Priority review pathways shorten approval review times to six months.
Yajun Ma
yajun.ma@lushmedia.com.au
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