Regulatory Index News: 08/05/2018

Welcome to your Regulatory Index News digest this Tuesday. Take a look here for reports of a new US approval, a negative CHMP review and a clinical halt for high doses.

Portola wins US approval for its coagulation factor, Andexxa

Andexxa, after several years of setbacks and delays, has won its US approval as a Factor Xa inhibitor reversal agent. The drug acts as a reversal agent to the new Factor Xa inhibitors Eliquis and Xarelto in that it reverses their blood-thinning effects for use in cases of uncontrolled bleeding or life-threatening situations. This comes as great news to those patients in the US, where there have been 115,000 cases reported of factor Xa inhibitor-related hospitalisations. However, the approval does carry a black box warning for thromembolic risks, ischemic risks, cardiac arrests and sudden death which highlights the risks of undoing blood-thinner induced anticoagulation. Portola Pharmaceuticals now looks to launch the drug on a limited basis this June followed by a complete roll-out by 2019. If you would like to read more, click here for a BioPharmaDIVE article.

Negative CHMP review for Sarepta’s Exondys

Sarepta have informed that there may be delays in the EU approval and launch of its Duchenne muscular dystrophy therapy, Exondys, after the CHMP voted negatively for its approval. The negative review is reported to be based on a lack of regulatory threshold for conditional approval, largely thanks to the use of external controls as study comparators. Sarepta are now planning to apply for re-examination and will request for a Scientific Advisory Group (SAG) to be convened to provide insight into the validity of external controls. The treatment itself is for those DMD patients with an exon 51 skipping mutation in their dystrophin gene (13% of DMD population) and is already conditionally approved in the US. Please click here for more from PharmaTimes.

Regeneron halts high dose fasinumab in phase 3

Regeneron have halted use of its osteoarthritis drug, fasinumab, at higher doses on the recommendation of its data monitoring committee after assessment of the treatments benefit-risk profile. The phase 3 trials where this has taken effect were testing the anti-NGF antibody in patients with osteoarthritis of the knee or hip as well as chronic low back pain. The issues experienced have been found before with other anti-NGF antibodies where arthritis is accelerated which can lead to increased joint replacements. Whilst it is not clear why this happens; one theory is that the therapy is so effective that the pain-free patients use their joints more causing increased deterioration. Now these higher-doses have been taken off the roster, Regeneron will narrow its focus on the lower doses which has been signed off by the data monitoring committee. Please click here for further information in a FiercePharma article.



Max Lymbery

Date Published

08th May 2018

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