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Alnylam’s amyloidosis drug heads latest crop of CHMP approvals

Three new biosimilars also gets the green light

EMA

The EMA’s main advisory committee backed the approval of 16 drugs at its meeting ahead of the weekend, including Alnylam’s patisiran for hereditary transthyretin-mediated (hATTR) amyloidosis.

The green light sets up the first product approval for Alnylam, an RNA interference specialist which has been working on gene-silencing drugs for around 15 years and is also due to hear from the US FDA on patisiran next month. If approved by the European Commission it will be sold as Onpattro.

The Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion for patisiran for the treatment of hATTR amyloidosis in adults who have developed peripheral nerve damage as a result of the  disease, which is characterised by the accumulation of amyloid in the nerves, heart and gastrointestinal tract, as well as other tissues.

The disease is caused by a defective gene called TTR that results in misfolded proteins that clump together as amyloid fibrils, and patisiran is designed to bind to and halt the RNA process that results in TTR protein expression. It’s the second drug for hATTR amyloidosis to clear the CHMP after Akcea/Ionis’ antisense oligonucleotide Tegsedi (inotersen), which was given a positive opinion in May and approved by the EC earlier this month.

The EMA said in a statement that having two drugs “considerably widens” the therapeutic options for hATTR amyloidosis, which include liver transplant, Pfizer’s Vyndaqel (tafamidis) and off-label non-steroidal anti-inflammatory drugs but have “considerable limitations” for patients with peripheral nerve damage.

Vertex’s new CF combo

There was also a thumbs up from the CHMP for Vertex’s latest cystic fibrosis drug Symkevi (ivacaftor/tezacaftor), a follow-up to its Kalydeco (ivacaftor) and Orkambi (ivacaftor/lumacaftor) products that are in the midst of a funding impasse in the UK. The drug was approved in the US as Symdeco in February.

Symkevi extends Vertex’s fast-growing range of exon-skipping therapies for CF, aimed at providing an option for all patients, regardless of the underlying mutation behind the disease. It has greater efficacy and is more tolerable than Orkambi, which has respiratory side effects that affect around a quarter of patients and can lead to therapy drop-outs.

The CHMP also recommended approval for four cancer drugs, including AstraZeneca’s immuno-oncology drugs Imfinzi (durvalumab) to prevent relapse after chemotherapy in non-small cell lung cancer (NSCLC) patients, billed as a potential blockbuster indication for the product.

It also backed three targeted cancer drugs: Pierre Fabre’s BRAF inhibitor Braftovi (encorafenib) and MEK inhibitor Mektovi (binimetinib) as a combination for BRAF V600-positive melanoma; and Eli Lilly’s CDK4/6 inhibitor Verzenios (abemaciclib) for hormone receptor-positive, HER2-negative breast cancer, already approved in the US (as Verzenio) and trying to chase down Pfizer’s category leader Ibrance (palbociclib).

Tetraphase Pharma got the go-ahead for Xerava (eravacycline), a new fluorocycline antibiotic for complicated intra-abdominal infections (cIAI), giving it some relief after some knockbacks with the drug, including a failed trial in complicated urinary tract infections (cUTI) that pitted the drug against Merck & Co/MSD’s Infanz (ertapenem). It filed for approval of the antibiotic in the US in January.

There was also good news for Almirall, which got the nod for its delayed interleukin-23 inhibitor Ilumetri (tildrakizumab) as a treatment for moderate to severe plaque psoriasis, although it enters an increasingly competitive field with new generation of biologics eating into a market previously dominated by TNF inhibitors. The drug was originally developed by India’s Sun Pharma and is licensed to Merck & Co in the US, where it was approved in March.

Paediatric use drugs

During its four-day meeting, the CHMP approved two new paediatric-use marketing authorisations (PUMAs) for off-patent drugs specifically developed for use in children. Currently, children and infants often have to be treated off-label with adult medicines as there are no approved drugs for them, for example by cutting down larger tablets that can give inaccurate dosing.

The first of the new products is RAD Neurim Pharma’s Slenyto, a small-tablet formulation of the hormone melatonin for the treatment of insomnia in children and adolescents with autism spectrum disorder or Smith-Magenis syndrome. There are currently no approved medicines to treat insomnia in children in the EU, although drugs like melatonin are used off-label.

Kigabeq (vigabatrin), the other PUMA medicine, has been developed by Orphelia Pharma for infantile spasms (West's syndrome) and resistant partial epilepsy (focal onset seizures) in infants and children. The product is available as 100mg to 500mg soluble tablets, in 50mg interim doses, to avoid having to cut down tablets or dilute oral solutions intended for adults.

Finally, the slow but steady stream of new biosimilars heading to market in Europe continues, with three new products recommended for approval that could provide cheaper alternatives to branded drugs.

The three biosimilars are: Mylan’s Hulio (adalimumab), a version of AbbVie’s big-selling Humira for the treatment of certain inflammatory and autoimmune disorders; and Accord Healthcare’s Pelgraz (pegfilgrastim) and ERA Consulting’s Udenyca (pegfilgrastim), both alternatives to Amgen’s Neulasta  brand for neutropenia and febrile neutropenia due to chemotherapy.

Article by
Phil Taylor

30th July 2018

From: Regulatory

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