Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 January 2018

Seven medicines recommended for approval, including one orphan

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended seven medicines for approval, including one orphan medicine¹, at its January 2018 meeting.

The CHMP recommended granting a marketing authorisation for Hemlibra (emicizumab), a first-in-class medicine to prevent bleeding episodes in patients with haemophilia A who have factor VIII inhibitors. This medicine was reviewed under EMA's accelerated assessment procedure, reserved for medicines of major public health interest. For more information, please see the press release in the grid below.

The CHMP recommended granting a marketing authorisation for Lamzede (velmanase alfa), a long-term enzyme replacement therapy in adults, adolescents and children with mild to moderate forms of alpha-mannosidosis. Because alpha-mannosidosis is a very rare disease, Lamzede was granted an orphan designation. For more information, please see the press release in the grid below.

The Committee recommended granting a marketing authorisation for Shingrix (recombinant, adjuvanted Herpes zoster vaccine), a vaccine for the prevention of herpes zoster and post-herpetic neuralgia in adults 50 years of age or older.

The CHMP gave positive opinions for three medicines to treat type 2 diabetes: Segluromet (ertugliflozin / metformin), Steglatro (ertugliflozin) and Steglujan (ertugliflozin / sitagliptin).

One biosimilar medicine was recommended for approval by the Committee: Semglee (insulin glargine), for the treatment of diabetes.

Re-adoption of opinion on new medicine

The CHMP confirmed its previous positive opinion and recommended the granting of a marketing authorisation for Lokelma (sodium zirconium cyclosilicate), for the treatment of hyperkalaemia.

Lokelma had received a positive opinion in February 2017. However, the European Commission suspended its decision-making following some concerns relating to good manufacturing practices (GMP) at the manufacturing site for the active substance and referred the matter back to the CHMP. A new inspection of the manufacturing site for Lokelma's active substance showed that the site is compliant with GMP. On the basis of the inspection findings, the Committee confirmed its previous opinion.

Negative opinion on new medicine

The CHMP adopted a negative opinion for EnCyzix (enclomifene). EnCyzix was expected to be used to treat hypogonadotrophic hypogonadism in men. For more information on this negative opinion, please see the questions-and-answers document in the grid below.

Start of re-examination of recommendation on new medicine

The applicant for Aplidin (plitidepsin) has requested a re-examination of the Committee's negative opinion for this medicine adopted at the December 2017 meeting. The CHMP will now re-examine this opinion and issue a final recommendation.

For more information on this negative opinion, please see the questions-and-answers document in the grid below.

Three recommendations on extensions of therapeutic indication

The Committee recommended extensions of indications for Hizentra, Relvar Ellipta and Revinty Ellipta.

Negative recommendation on extension of indication following re-examination

The applicant for Raxone (idebenone) requested a re-examination of the Committee's negative opinion for this medicine adopted at the September 2017 meeting. After considering the grounds for this request, the CHMP re-examined the opinion, and confirmed the refusal of the change to the marketing authorisation.

For more information on this negative opinion, please see the questions-and-answers document in the grid below.

Withdrawals of applications

Applications for initial marketing authorisations for Balimek (binimetinib) and Rotigotine Mylan (rotigotine) have been withdrawn. Balimek was intended to be used to treat melanoma (a type of skin cancer) that had spread or could not be removed by surgery. Rotigotine Mylan was intended to be used to treat Parkinson's disease and restless-leg syndrome.

An application to extend the use of Opdivo (nivolumab) to treat colorectal cancer has also been withdrawn.

Questions-and-answers documents on these withdrawals are available in the grid below.

Agenda and minutes

The agenda of the January 2018 meeting is published on EMA's website. Minutes of the December 2017 CHMP meeting will be published in the coming weeks.

CHMP statistics

Key figures from the January 2018 CHMP meeting are represented in the graphic below.

More information on all other outcomes of the CHMP January 2018 meeting is available in the grid below.

¹As always at time of approval, this orphan designation will now be reviewed by EMA's Committee for Orphan Medicinal Products (COMP) to determine whether the information available to date allows maintaining the medicine's orphan status and granting the medicine ten years of market exclusivity.

CHMP statistics: January 2018

Positive recommendations on new medicines

Name of medicine	Hemlibra
International non-proprietary name (INN)	emicizumab
Marketing-authorisation applicant	Roche Registration Limited
Therapeutic indication	Prevention of bleeding episodes in patients with haemophilia A who have factor VIII inhibitors
More information	CHMP summary of positive opinion for Hemlibra Press release: First-in-class medicine to prevent bleeding in haemophilia A patients with inhibitors

Name of medicine	Lamzede
INN	velmanase alfa
Marketing-authorisation applicant	Chiesi Farmaceutici S.p.A.
Therapeutic indication	Treatment of patients with non-neurological manifestations of mild to moderate alpha-mannosidosis
More information	CHMP summary of positive opinion for Lamzede Press release: New enzyme replacement therapy to treat rare genetic disorder alpha-mannosidosis in children and adults

Name of medicine	Segluromet
INN	ertugliflozin / metformin
Marketing-authorisation applicant	Merck Sharp & Dohme Limited
Therapeutic indication	Treatment of type 2 diabetes
More information	CHMP summary of positive opinion for Segluromet

Name of medicine	Shingrix
Common name	herpes zoster vaccine (recombinant, adjuvanted)
Marketing-authorisation applicant	GlaxoSmithkline Biologicals SA
Therapeutic indication	Prevention of herpes zoster (HZ) and post-herpetic neuralgia (PHN), in adults 50 years of age or older
More information	CHMP summary of positive opinion for Shingrix

Name of medicine	Steglatro
INN	ertugliflozin
Marketing-authorisation applicant	Merck Sharp & Dohme Limited
Therapeutic indication	Treatment of type 2 diabetes
More information	CHMP summary of positive opinion for Steglatro

Name of medicine	Steglujan
INN	ertugliflozin / sitagliptin
Marketing-authorisation applicant	Merck Sharp & Dohme Limited
Therapeutic indication	Treatment of type 2 diabetes
More information	CHMP summary of positive opinion for Steglujan

Positive recommendation on new biosimilar medicine

Name of medicine	Semglee
INN	insulin glargine
Marketing-authorisation applicant	Mylan S.A.S
Therapeutic indication	Treatment of diabetes
More information	CHMP summary of positive opinion for Semglee

Re-adoption of opinion for new medicine

Name of medicine	Lokelma
INN	sodium zirconium cyclosilicate
Marketing-authorisation applicant	AstraZeneca AB
Therapeutic indication	Treatment of hyperkalaemia
More information	CHMP summary of positive opinion for Lokelma

Negative recommendation for new medicine

Name of medicine	EnCyzix
INN	enclomifene
Marketing-authorisation applicant	Renable Pharma Limited
Therapeutic indication	Treatment of hypogonadotropic hypogonadism in men
More information	Questions and answers on refusal of the marketing authorisation for EnCyzix (enclomifene)

Start of re-examination of recommendation on new medicine

Name of medicine	Aplidin
INN	plitidepsin
Marketing-authorisation applicant	Pharma Mar
Therapeutic indication	Treatment of multiple myeloma
More information

Positive recommendations on extensions of indications

Name of medicine	Hizentra
INN	human normal immunoglobulin
Marketing-authorisation holder	CSL Behring GmbH
More information	CHMP post-authorisation summary of positive opinion for Hizentra (II-0087)

Name of medicine	Relvar Ellipta
INN	fluticasone furoate / vilanterol
Marketing-authorisation holder	Glaxo Group Ltd
More information	CHMP post-authorisation summary of positive opinion for Relvar Ellipta (WS-1208)

Name of medicine	Revinty Ellipta
INN	fluticasone furoate / vilanterol
Marketing-authorisation holder	Glaxo Group Ltd
More information	CHMP post-authorisation summary of positive opinion for Revinty Ellipta

Negative recommendation on extension of indication following re-examination

Name of medicine	Raxone
INN	idebenone
Marketing-authorisation holder	Santhera Pharmaceuticals (Deutschland) GmbH
More information	Questions and answers on the refusal of a change to the marketing authorisation for Raxone (idebenone)

Withdrawals of initial marketing autorisation applications

Name of medicine	Balimek
INN	binimetinib
Marketing-authorisation applicant	Pierre Fabre Médicament
More information	Questions and answers on the withdrawal of the marketing authorisation application for Balimek (binimetinib)

Name of medicine	Rotigotine Mylan
INN	rotigotine
Marketing-authorisation applicant	Mylan S.A.S
More information	Questions and answers on withdrawal of the marketing authorisation application for Rotigotine Mylan (rotigotine)

Withdrawal of extension of indication application

Name of medicine	Opdivo
INN	nivolumab
Marketing-authorisation holder	Bristol-Myers Squibb Pharma EEIG
More information	Questions and answers on the withdrawal of the application for a change to the marketing authorisation for Opdivo (nivolumab)

Other updates

Recommendations on eligibility to PRIME scheme - Adopted at the CHMP meeting of 22-25 January 2018

Scientific advice and protocol assistance adopted during the CHMP meeting 22 – 25 January 2018

Overview of (invented) names reviewed in December 2017 by the Name Review Group (NRG)