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Thirty orphan drugs face status lapse
Posted 10 October 2017
New TGA reforms will see orphan drug designations granted before July 2017 lapse in the next 12 months, putting at least 30 current drugs on track to designation expiry unless they are filed before then.
According to Pharma in Focus research, at least 30 drugs with orphan designations granted since 2015 are still unregistered on the ARTG while a further eight have unregistered extended indications.
These will need to be filed in the next 12 months to receive the ARTG fee waiver which comes with orphan drug designation. With fees for ARTG registration coming in at $231,000 according to a recent comparison of global regulators, up to $7 million in savings is on the line if sponsors fail to act in time.
The deadline has been implemented as part of a transition process allowing sponsors of drugs designated before July 2017 to adjust to new reforms which have put a stop to the previously indefinite period allowed for orphan drug status.
Now sponsors must lodge an ARTG application within six months of being granted orphan drug status before it expires "to ensure that the fee waiver of a related registration application is based on information that is reasonably current," although a further six-month extension is possible "in certain circumstances".
Among the list of drugs granted orphan status but still not ARTG registered, most were listed in 2016 and 2017 with some still lingering on from years further back including AstraZeneca's tremelimumab for the treatment of mesothelioma, which has been on the designation list since November 2015. The drug is not yet approved in any jurisdiction.
Other drugs on the list which are yet to see global approvals include MSD's transplant treatment letermovir, GSK's malaria drug tafenoquine and AbbVie's Rova-T (rovalpituzumab tesirine) for small cell lung cancer.
Last year blood cancer drugs Merck Serono's Bavencio (avelumab) and Pfizer's Besponsa (inotuzumab ozogamicin) were also designated. Both drugs have received approvals in the US and EU this year. Takeda's lung cancer drug Alunbrig (brigatinib), which won an accelerated approval in the US last month, was one of the most recent designations.
Drugs listed for orphan extensions of indications include Eisai's Lenvima (lenvantinib) for a liver cancer indication, GSK's Nucala (mepolizumab) for the treatment of eosinophilic granulomatosis with polyangiitis and Alexion's Soliris (eculizumab) for prevention of delayed graft function after solid transplant.
Given most orphan drugs are registered within two years of designation, the drugs are most likely filed within a year of designation meaning a large portion of the current unregistered list will make the 2018 deadline.
The new reforms are the first in 20 years to affect the orphan drug program.
Yajun Ma
yajun.ma@lushmedia.com.au
Posted 10 October 2017
New TGA reforms will see orphan drug designations granted before July 2017 lapse in the next 12 months, putting at least 30 current drugs on track to designation expiry unless they are filed before then.
According to Pharma in Focus research, at least 30 drugs with orphan designations granted since 2015 are still unregistered on the ARTG while a further eight have unregistered extended indications.
These will need to be filed in the next 12 months to receive the ARTG fee waiver which comes with orphan drug designation. With fees for ARTG registration coming in at $231,000 according to a recent comparison of global regulators, up to $7 million in savings is on the line if sponsors fail to act in time.
The deadline has been implemented as part of a transition process allowing sponsors of drugs designated before July 2017 to adjust to new reforms which have put a stop to the previously indefinite period allowed for orphan drug status.
Now sponsors must lodge an ARTG application within six months of being granted orphan drug status before it expires "to ensure that the fee waiver of a related registration application is based on information that is reasonably current," although a further six-month extension is possible "in certain circumstances".
Among the list of drugs granted orphan status but still not ARTG registered, most were listed in 2016 and 2017 with some still lingering on from years further back including AstraZeneca's tremelimumab for the treatment of mesothelioma, which has been on the designation list since November 2015. The drug is not yet approved in any jurisdiction.
Other drugs on the list which are yet to see global approvals include MSD's transplant treatment letermovir, GSK's malaria drug tafenoquine and AbbVie's Rova-T (rovalpituzumab tesirine) for small cell lung cancer.
Last year blood cancer drugs Merck Serono's Bavencio (avelumab) and Pfizer's Besponsa (inotuzumab ozogamicin) were also designated. Both drugs have received approvals in the US and EU this year. Takeda's lung cancer drug Alunbrig (brigatinib), which won an accelerated approval in the US last month, was one of the most recent designations.
Drugs listed for orphan extensions of indications include Eisai's Lenvima (lenvantinib) for a liver cancer indication, GSK's Nucala (mepolizumab) for the treatment of eosinophilic granulomatosis with polyangiitis and Alexion's Soliris (eculizumab) for prevention of delayed graft function after solid transplant.
Given most orphan drugs are registered within two years of designation, the drugs are most likely filed within a year of designation meaning a large portion of the current unregistered list will make the 2018 deadline.
The new reforms are the first in 20 years to affect the orphan drug program.
Yajun Ma
yajun.ma@lushmedia.com.au
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