UPDATED: Tuesday, December 19 at 8:40 a.m.

• The number of approved indications for Merck's star cancer medicine Keytruda has grown to 26 in Europe.
  
  Most recently, the European Commission approved the drug—in two chemotherapy combinations—for separate first-line uses in gastrointestinal cancers.
  
  The first approval covers the use of Keytruda, plus fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma in certain adults whose tumors express PD-L1.
  
  Additionally, the EC signed off on Keytruda's use in combination with gemcitabine and cisplatin for the first-line treatment of adults with locally advanced unresectable or metastatic biliary tract carcinoma.
  
  Europe's Committee for Medicinal Products for Human Use (CHMP) previously endorsed these uses based on data from Merck's KEYNOTE-859 and KEYNOTE-966 studies, respectively.
   
• Also in Europe, Biogen said that the CHMP granted a positive opinion for Skyclarys to treat Friedreich’s ataxia in people 16 and older.
  
  If the EC signs off on the drug's approval as expected, Skyclarys would be the first Friedreich’s ataxia medicine in Europe. 
  
  In a 48-week study called MOXIe Part 2, patients who received the drug had less physical impairment, as measured by the modified Friedreich Ataxia Rating Scale, than those who received placebo.
  
  Biogen picked up Skyclarys, a potential blockbuster, when it acquired Reata Pharmaceuticals earlier this year.
   
• Lastly, Ultragenyx's treatment for an ultrarare, inherited form of high cholesterol won an expanded approval from the European Commission.
  
  The EC endorsed Evkeeza as an adjunct to diet and other medicine to treat children ages 5 to 11 with homozygous familial hypercholesterolemia. The approval builds on the medicine's first nod in patients 12 and older back in 2021.
   

UPDATED: Friday, December 15 at 10:15 a.m.

• Following approvals in the U.K. and the U.S., Vertex's CRISPR-based gene-editing therapy Casgevy has picked up a positive opinion from the European Medicines Agency (EMA). 
  
  The European agency recommends an approval for Casgevy for transfusion‑dependent beta thalassemia and severe sickle cell disease in patients 12 years of age and older who are eligible for a stem cell transplant but who don't have a suitable donor.
  
  The EMA reviewed Casgevy's application under its priority medicines scheme, and the therapy is recommended for a conditional marketing authorization. Once Casgevy is approved by the European Commission, Vertex is on the hook to submit the final results from its pivotal trials by August 2026, as well as results from an ongoing long-term follow-up study, among other data. Patients who got Casgevy will be followed up for 15 years.
   
• Despite GSK's appeal, the EMA's Committee for Medicinal Products for Human Use decided not to renew a conditional approval for the company's multiple myeloma drug Blenrep. 
  
  The EU regulator based its decision on findings from the DREAMM-3 trial, which showed that Blenrep couldn't outdo Bristol Myers Squibb's Pomalyst and dexamethasone at slowing disease progression or death in previously treated patients. GSK last year pulled Blenrep from the U.S. market because of the same flop.
  
  The EMA will now send its opinion to the European Commission for a final decision. Once the decision is formally adopted as expected in Feburary 2024, GSK will work with the EU member states to initiate withdrawal of Blenrep, a GSK spokesperson told Fierce Pharma.
  
  The regulatory setback doesn't affect GSK's plan for Blenrep in other combinations, the spokesperon said. The company recently announced positive findings from the DREAMM-7 trial, showing Blenrep extended progression-free survival compared with Johnson & Johnson’s Darzalex in their respective combinations with Takeda’s Velcade and the steroid dexamethasone in multiple myeloma patients who had tried at least one prior line of treatment.
   
• In addition to Casgevy, the EMA also recommended approvals for Pfizer's Velsipity in moderately to severely active ulcerative colitis, Biogen/Reata's Skyclarys for Friedreich’s ataxia and Takeda's HyQvia as a maintenance therapy for chronic inflammatory demyelinating polyneuropathy.
  
  The agency also expanded its recommendation for Sanofi's Fexinidazole Winthrop to treat Trypanosoma brucei (T.b.) rhodesiense sleeping sickness. Years ago, the drug was recommended for the more common T.b. gambiense form of sleeping sickness found in West and Central Africa.
   

UPDATED: Thursday, December 14 at 8:55 a.m.

• After a recent meeting with European regulators, Apellis said it has been "informed of a negative trend vote" on its filing for pegcetacoplan to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD). 
  
  In a Thursday update, Apellis said it expects Europe's Committee for Medicinal Products for Human Use to "adopt a negative opinion" on the application.
  
  If that happens, Apellis said it will appeal and seek a re-examination of the filing. That process could take 4 to 5 months, Mizuho analysts said in a Thursday note.
  
  “We are very disappointed in this outcome and saddened for the millions of GA patients in Europe who have no treatment for this leading cause of blindness,” Apellis' chief development officer, Jeffrey Eisele, Ph.D., said in a statement. 
  
  Apellis is leveraging its phase 3 Oaks and Derby studies for the filing. The drug picked up its FDA approval in GA back in February.
   
• In the U.S., Michigan-based drugmaker Esperion has received several label updates for its cholesterol-busting drugs Nexletol and Nexlizet.
  
  Thanks to FDA nods in 2020 and 2021, the drugs have been approved to treat patients with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular disease who require additional lowering of LDL-C.
  
  In a recent update, the FDA added primary hyperlipidemia as a qualifier for treatment in the approved patient populations. Further, the agency removed a requirement that patients try a "maximally tolerated" statin dose before starting on the Esperion drugs.
  
  Esperion is still aiming for a label addition detailing the drugs' cardiovascular risk reduction, with a decision on that filing expected by the end of March. The company also expects European approval to come in the first half of 2024.
   

UPDATED: Monday, December 11 at 9:55 a.m.

• GSK's PD-1 cancer drug Jemperli has picked up a European approval to treat adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer who are candidates for systemic therapy. The approval covers the use of Jemperli in combination with carboplatin-paclitaxel in this group of patients.
   
  With the nod, Jemperli becomes the first frontline immuno-oncology treatment for this patient population in Europe, GSK said in a Monday release.
  
  Besides the frontline approval, the European Commission converted a prior conditional second-line approval in dMMR/MSI-H recurrent or advanced endometrial cancer into a full nod.
  
  Results from the phase 3 Ruby study enabled the new approval. In the study, investigators noted a 72% reduction in the risk of disease progression or death for the Jemperli regimen compared with chemotherapy alone.
  
  "With this approval, we can expand the number of patients who can potentially benefit from treatment with Jemperli in Europe, including patients who are earlier in their journey," GSK's global oncology R&D head, Hesham Abdullah, said in a statement.
   
• Also in Europe, Astellas' Veoza has gained regulatory approval to treat moderate to severe vasomotor symptoms (VMS) associated with menopause.
  
  The approval follows an October recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in favor of a marketing authorization. More than 3,000 participants took part in phase 3 trials enabling the regulatory nod.
  
  In the U.S., the drug won its FDA nod back in May. It's marketed as Veozah in the States.