BioCryst’s Orladeyo (berotralstat) has received a positive recommendation from the National Institute for Health and Care Excellence (NICE) for preventing recurrent attacks of hereditary angioedema (HAE) in people aged 12 years and older.

In its draft guidance, NICE has recommended the treatment for people who have at least two attacks per month. Orladeyo treatment should be stopped if the number of attacks per month does not reduce by at least 50% after three months of use.

HAE affects around one person per 50,000 to 100,000, and is characterised as a rare genetic disorder.

People living with this condition experience unpredictable and recurrent attacks of swelling in the mouth, gut or airway. In turn, this can lead to breathing difficulties and severe pain and can also cause swelling in multiple body parts.

Clinical trial evidence for Orladeyo suggests that the treatment reduces the frequency of swelling attacks, although the effect on the severity of these attacks is ‘uncertain’, according to NICE.

The Scottish Medicines Consortium (SMC) has accepted three new medicines as part of its September 2021 decisions.

This includes Eli Lilly's Retsevmo (selpercatinib), which has been accepted for interim use subject to ongoing evaluation and future reassessment, for the treatment of rare RET fusion-positive thyroid cancers. Specifically, Retsevmo is indicated for use in patients who require systemic therapy following prior treatment with sorafenib and/or lenvatinib. In a Phase I/II study in previously treated patients with advanced RET-mutant thyroid cancer or RET-mutant medullary thyroid cancer (MTC), Retsevmo was associated with an objective response rate of 79% and 69% respectively.
  
Meanwhile, MSD’s Keytruda (pembrolizumab) has been accepted for the first-line treatment of metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer in adults.

In addition, Gilead and Galapagos' Jyseleca (filgotinib) has been accepted for the treatment of severe rheumatoid arthritis where other treatments have failed or could not be tolerated.

In a statement, the SMC said Jyseleca provides another oral treatment option for patients with this condition, and may help to delay disease progression and prolong the time that patients are able to continue working and living independently.

Amgen has announced that its first-in-class KRAS G12C inhibitor Lumykras (sotorasib) has received a conditional marketing authorisation in the UK.

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has conditionally approved Lumykras under Project Orbis – an international collaborative programme between the US Food and Drug Administration and regulatory agencies across the world.

KRAS G12C is one of the most common identified or known drivers in non-small cell lung cancer (NSCLC).

Amgen’s med binds with a mutated KRAS G12C protein in a bid to ‘switch off’ the signals it sends to trigger cell division and cancer cell growth.

The MHRA review of Lumykras considered data from the Phase II CodeBreaK 100 clinical study, which evaluated the drug in 126 patients with KRAS G12c-mutated advanced NSCLC.

Results from this trial demonstrated a confirmed objective response rate (ORR) of 37.1% and a disease control rate (DCR) of 80.6% for Lumykras-treated patients.
Recently published data also showed a median overall survival (OS) of 12.5 months among 124 evaluable patients, with the median duration of response (DoR) – evaluated in 46 patients – shown to be 11.1 months.