The US Food and Drug Administration (FDA) has approved PTC Therapeutics’ oral drug to treat a rare genetic disorder known as phenylketonuria (PKU). The agency also okayed Apellis Pharmaceutical’s Empaveli to treat two rare kidney disorders.There was news about
two likely buyouts. First, a consortium led by Nordic Capital and Permira have made an offer of around US$ 3 billion to buy vaccine maker Bavarian Nordic. And a Bloomberg report said AbbVie is in talks to acquire privately-owned mental health therapeutics company Gilgamesh Pharmaceuticals for around US$ 1 billion.In deals, GSK signed an up to US$ 12 billion biobucks deal with China’s Hengrui Pharma to work on up to 12 drugs in the respiratory, immunology, inflammation and oncology space. Novartis entered into an exclusive deal with Matchpoint Therapeutics to develop drugs for a number of inflammatory diseases.Madrigal Pharmaceuticals signed a licensing deal with China’s CSPC Pharma Group for a liver disease drug. And Boehringer Ingelheim and Re-Vana Therapeutics announced an agreement to
develop therapies for eye diseases.In a head-to-head study, Eli Lilly’s blood cancer drug Jaypirca (pirtobrutinib) was found to be more effective than AbbVie’s Imbruvica. And AstraZeneca’s experimental therapy, gefurulimab, met the primary
goal and all secondary endpoints in a late-stage study aimed at improving symptoms of
generalized myasthenia Gravis (gMG), a rare autoimmune condition.In other news, the European Commission signed a trade deal with the US, which is likely to result in higher drug prices for Americans. And Vinay Prasad, FDA’s top vaccine official, has quit just three months after joining as the Director of the Center for Biologics Evaluation and Research (CBER).FDA approves PTC Therapeutics’ rare genetic disorder drugThe US Food and Drug Administration (FDA)
has approved PTC Therapeutics’ oral drug — Sephience (sepiapterin) — to treat a rare genetic disorder known as phenylketonuria (PKU). Sephience is an oral therapy for both children and adults with PKU. In PKU, the body can’t properly break down an amino acid known as phenylalanine, leading to its
buildup in the blood which can potentially damage the brain. The approval is based on the evidence of significant efficacy and safety from a phase 3
trial.Apellis’ med okayed for two rare kidney diseases: FDA has approved Apellis Pharmaceuticals’ Empaveli (pegcetacoplan) as the first treatment for two rare kidney diseases known as C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis.Nordic Capital, Permira make
US$ 3 billion offer for vaccine maker Bavarian NordicVaccine maker Bavarian Nordic has announced
that a consortium led by Nordic Capital and Permira has made an offer of around US$ 3 billion to acquire it. The offer
price marks a 21 percent premium over Bavarian Nordic’s stock closing price of July 23, 2025. According to a press statement, the board of Bavarian Nordic has found the offer to be attractive, and has recommended that the “Bavarian Nordic shareholders accept” the offer when made.GSK signs US$ 12 bn biobucks deal with Hengrui: GSK has signed an up to US$ 12 billion biobucks deal with China’s Hengrui Pharma to work on up to 12 drugs. GSK is paying Hengrui US$ 500 million
upfront. The programs were selected to complement GSK’s extensive respiratory, immunology, inflammation and oncology pipelines.Madrigal in deal with CSPC to develop liver drug: American drugmaker Madrigal Pharmaceuticals has signed a licensing deal with China’s CSPC Pharma Group for a liver disease drug. The deal could be potentially worth US$ 2 billion.Novartis in US$ 1 bn deal with Matchpoint; AbbVie may acquire GilgameshMatchpoint Therapeutics, a biotechnology
company, has entered into an exclusive option and license agreement with Novartis for the development and commercialization of several oral drugs to
treat various inflammatory diseases. Matchpoint is likely to receive up to US$
60 million in upfront payment and research funding, with up to US$ 1 billion in total potential
payments.AbbVie in talks to buy Gilgamesh: AbbVie is in talks to acquire privately-owned
mental health therapeutics company Gilgamesh Pharmaceuticals in a deal that could be
worth about US$ 1 billion, a
Bloomberg news report said.Boehringer signs deal with Re-Vana: Boehringer Ingelheim and Re-Vana Therapeutics have announced a strategic collaboration and license
agreement that aims to develop first-in-class extended-release therapies for eye diseases.Europe-US trade deal could result in higher drug prices for AmericansOn 27 July 2025, European Commission
President Ursula von der Leyen and US President Donald Trump agreed on a trade deal. This deal is said to cost
the pharmaceutical industry between US$ 13 billion and US$ 19 billion as branded medicines become subject to a tariff of 15 percent. Though this tariff rate is much lower than the levies of up to 200
percent that Trump had threatened Europe with, it stands to add billions of
dollars in expenses for the drug industry and could lead to price increases for
some medicines.FDA’s top vaccine official resigns: Vinay Prasad, FDA’s top vaccine official, has left the agency after just three months of joining it as the Director of the Center for Biologics Evaluation and Research (CBER). Of late, Prasad had faced criticism for the agency’s handling of Sarepta Therapeutics’ gene therapy for Duchenne
muscular dystrophy. Prasad, was also a fierce critic of America’s Covid vaccine and mask mandates. George Tidmarsh, who was recently appointed as head of FDA’s Center for Drug Evaluation and Research, will also be the acting CBER director.Lilly’s leukemia med Jaypirca beats AbbVie’s Imbruvica in head-to-head trialIn a
head-to-head study, Eli Lilly’s blood cancer drug Jaypirca (pirtobrutinib) was found to be more effective than AbbVie’s Imbruvica (ibrutinib) when tested in both untreated and previously treated patients. Jaypirca was tested in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma, two kinds of blood cancer.Astra’s rare disease drug scores trial win: AstraZeneca experimental therapy, gefurulimab, met the primary goal and all secondary
endpoints in a late-stage study aimed at improving symptoms of generalized myasthenia Gravis (gMG), a
rare autoimmune condition that affects muscle function. The therapy reduced the
severity of gMG in adults and improved functional activities, when compared
with placebo at 26 weeks.Meanwhile, Alfasigma’s Jyseleca (filgotinib) met its primary endpoint in
treating adults with active axial
spondyloarthritis (axSpA), a type of inflammatory arthritis, in a phase 3
trial. Filgotinib is currently approved for the treatment of moderate to severe active rheumatoid arthritis (RA) and ulcerative colitis (UC).
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